Latest news with #MyastheniaGravis
Globe and Mail
18-06-2025
- Business
- Globe and Mail
Sanofi, Cartesian Therapeutics, Takeda, UCB Biopharma, Hoffmann-La Roche, Janssen Research & Development, LLC
The Key Myasthenia Gravis Companies in the market include - Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Sanofi, Cartesian Therapeutics, Takeda, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, DAS Therapeutics, Chugai Pharmaceutical, Inc., Alexion, Regeneron Pharmaceuticals, Ra Pharmaceuticals, Inc., and others. DelveInsight's 'Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Myasthenia Gravis, historical and forecasted epidemiology as well as the Myasthenia Gravis market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan. Some of the key facts of the Myasthenia Gravis Market Report: Among the 7MM countries the Myasthenia Gravis market size was valued approximately USD 3,880 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034) In April 2025, Impressive findings from the Phase 3 clinical trial (NCT05737160) evaluating the safety and effectiveness of Telitacicept (also known as RC18; brand name: 泰爱®) in individuals with generalized myasthenia gravis (gMG) were presented during the Late-Breaking Science Session at the 2025 American Academy of Neurology (AAN) Annual Meeting. In March 2025, Immunovant has opted not to seek regulatory approval for its myasthenia gravis (MG) therapy, batoclimab, even though the Phase III trial achieved its primary goal of reducing disease symptoms. Instead, the company plans to use the trial data to support the advancement of its alternative candidate, IMVT-1402. The Phase III randomized study (NCT05403541) evaluated the effects of weekly or bi-weekly batoclimab dosing in acetylcholine receptor antibody-positive (AChR+) patients, measuring improvements through the Myasthenia Gravis Activities of Daily Living (MG-ADL) score over 12 weeks. In December 2024, Cartesian Therapeutics has shared updated efficacy and safety results from its Phase IIb trial evaluating Descartes-08, an mRNA cell therapy candidate for generalized myasthenia gravis (MG). The findings demonstrated durable responses at 12 months. This open-label, double-blind, placebo-controlled, crossover study (NCT06038474) enrolled 36 symptomatic patients who had previously undergone extensive treatment. Participants were divided evenly, with one group receiving a placebo and the other receiving Descartes-08. Both groups underwent six weekly outpatient infusions, administered without the need for prior chemotherapy-based preconditioning. In October 2024, Cartesian Therapeutics, Inc. (NASDAQ: RNAC), a clinical-stage biotechnology company pioneering mRNA cell therapies for autoimmune disorders, announced plans to present findings from its Phase 2b trial of Descartes-08 in patients with generalized myasthenia gravis (MG). The data will be shared at the 2024 Myasthenia Gravis Foundation of America (MGFA) Scientific Session, held during the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Annual Meeting. The combined myasthenia gravis market size in the European Union's four largest economies and the United Kingdom was approximately USD 950 million in 2023. This figure is expected to increase over the forecast period from 2024 to 2034. In June 2024, Johnson & Johnson (NYSE: JNJ) has announced encouraging outcomes from the Phase 3 Vivacity-MG3 trial of nipocalimab in patients with generalized myasthenia gravis (gMG). The trial demonstrated that patients receiving nipocalimab alongside standard of care (SOC) showed significant improvement in the MG-ADL score over 24 weeks compared to those on placebo plus SOC, meeting the primary endpoint. These findings are part of eight abstracts Johnson & Johnson will present at the 2024 European Academy of Neurology (EAN) Congress. Additionally, the data will be included in regulatory submissions later this year. In January 2024, Johnson & Johnson reported the headline findings from the critical Phase III VIVACITY trial of nipocalimab in adults with generalized myasthenia gravis (gMG). In this Phase III study, nipocalimab met its primary goal, demonstrating a statistically significant decrease in MG-ADL score from baseline during weeks 22 to 24 compared to placebo (PBO). According to DelveInsight's evaluation in 2023, the seven major markets (7MM) had an estimated 297,000 diagnosed prevalent instances of myasthenia gravis, with an expected upward trajectory in these cases anticipated during the forecast period from 2024 to 2034. In 2023, the United States had the largest number of diagnosed prevalent cases of myasthenia gravis among the seven major markets (7MM), totaling approximately 133,000 cases. It is projected that these figures will increase during the forecast period from 2024 to 2034. In 2023, myasthenia gravis affected 48% of males and 52% of females across the seven major markets (7MM). In the United States, there were approximately 66,000 cases among males and around 67,000 cases among females in 2023. These gender-specific incidences are projected to increase during the forecast period from 2024 to 2034. Emerging treatments for myasthenia gravis like Nipocalimab, Batoclimab, Descartes-08, ENSPRYNG (satralizumab), and others hold promise to substantially impact the market size of myasthenia gravis in a positive direction. Key Myasthenia Gravis Companies: Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Sanofi, Cartesian Therapeutics, Takeda, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, DAS Therapeutics, Chugai Pharmaceutical, Inc., Alexion, Regeneron Pharmaceuticals, Ra Pharmaceuticals, Inc., and others Key Myasthenia Gravis Therapies: Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Sanofi, Cartesian Therapeutics, Takeda, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, DAS Therapeutics, Chugai Pharmaceutical, Inc., Alexion, Regeneron Pharmaceuticals, Ra Pharmaceuticals, Inc., and others The Myasthenia Gravis market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Myasthenia Gravis pipeline products will significantly revolutionize the Myasthenia Gravis market dynamics. Myasthenia Gravis Overview Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disorder characterized by weakness and fatigue of voluntary muscles. It occurs when the body's immune system mistakenly attacks and damages the communication between nerve cells and muscles, specifically at the neuromuscular junction. Myasthenia Gravis Epidemiology The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends. Myasthenia Gravis Epidemiology Segmentation: The Myasthenia Gravis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalence of Myasthenia Gravis Prevalent Cases of Myasthenia Gravis by severity Gender-specific Prevalence of Myasthenia Gravis Diagnosed Cases of Episodic and Chronic Myasthenia Gravis Download the report to understand which factors are driving Myasthenia Gravis epidemiology trends @ Myasthenia Gravis Epidemiology Forecast Myasthenia Gravis Drugs Uptake and Pipeline Development Activities The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Myasthenia Gravis market or expected to get launched during the study period. The analysis covers Myasthenia Gravis market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share. The report also covers the Myasthenia Gravis Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies. Myasthenia Gravis Therapies and Key Companies Myasthenia Gravis Market Strengths The growing prevalence of myasthenia gravis (MG) and the geriatric population offers a stable market demand for treatments and therapies. Myasthenia gravis treatments may qualify for orphan drug status, offering regulatory incentives, such as extended market exclusivity and tax benefits. Myasthenia Gravis Market Opportunities Potential for developing new, more effective therapies, such as gene therapy or targeted biologics, could address unmet medical needs. Improved diagnostic techniques using clear biomarkers and an increase in awareness will likely lead to a diagnosis of more patients, expanding the market. Scope of the Myasthenia Gravis Market Report Study Period: 2020-2034 Coverage: 7MM Countries Key Myasthenia Gravis Companies: Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Sanofi, Cartesian Therapeutics, Takeda, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, DAS Therapeutics, Chugai Pharmaceutical, Inc., Alexion, Regeneron Pharmaceuticals, Ra Pharmaceuticals, Inc., and others Key Myasthenia Gravis Therapies: Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Sanofi, Cartesian Therapeutics, Takeda, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, DAS Therapeutics, Chugai Pharmaceutical, Inc., Alexion, Regeneron Pharmaceuticals, Ra Pharmaceuticals, Inc., and others Myasthenia Gravis Therapeutic Assessment: Myasthenia Gravis current marketed and Myasthenia Gravis emerging therapies Myasthenia Gravis Market Dynamics: Myasthenia Gravis market drivers and Myasthenia Gravis market barriers Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies Myasthenia Gravis Unmet Needs, KOL's views, Analyst's views, Myasthenia Gravis Market Access and Reimbursement Table of Contents 1. Myasthenia Gravis Market Report Introduction 2. Executive Summary for Myasthenia Gravis 3. SWOT analysis of Myasthenia Gravis 4. Myasthenia Gravis Patient Share (%) Overview at a Glance 5. Myasthenia Gravis Market Overview at a Glance 6. Myasthenia Gravis Disease Background and Overview 7. Myasthenia Gravis Epidemiology and Patient Population 8. Country-Specific Patient Population of Myasthenia Gravis 9. Myasthenia Gravis Current Treatment and Medical Practices 10. Myasthenia Gravis Unmet Needs 11. Myasthenia Gravis Emerging Therapies 12. Myasthenia Gravis Market Outlook 13. Country-Wise Myasthenia Gravis Market Analysis (2020-2034) 14. Myasthenia Gravis Market Access and Reimbursement of Therapies 15. Myasthenia Gravis Market Drivers 16. Myasthenia Gravis Market Barriers 17. Myasthenia Gravis Appendix 18. Myasthenia Gravis Report Methodology 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight About DelveInsight DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach. Media Contact Company Name: DelveInsight Contact Person: Gaurav Bora Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:
Yahoo
17-06-2025
- Business
- Yahoo
RemeGen's Telitacicept (RC18) Received Orphan Drug Designation from EMA for Myasthenia Gravis
YANTAI, China, June 17, 2025 /PRNewswire/ -- On June 16, 2025, RemeGen Co., Ltd. ("RemeGen", stock symbols: announced that telitacicept (RC18; brand name: 泰爱®) has received Orphan Drug Designation (ODD) from European Medicines Agency (EMA) for the treatment of Myasthenia Gravis (MG), marking a key milestone achieved in its global development. Telitacicept now is the first dual-target biologic drug for MG with ODD from both FDA and EMA worldwide. The designation, granted based on telitacicept's significant benefits in treating the life-threatening rare disease of MG, will provide advantages to this novel agent on protocol assistance, regulatory fee reductions/waivers and up to 10 years of market exclusivity, thereby accelerating the clinical development, registration and drug approval process in Europe and its availability among MG patients. MG is an acquired antibody-mediated rare autoimmune disorder affecting the neuromuscular junction. According to the Myasthenia Gravis Foundation of America (MGFA) and various studies, its global prevalence is estimated to be 15-25 per 100,000, meeting EMA's definition of the rare disease (the prevalence of less than 5 in 10,000 in the EU). Though the current treatment options (including cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulins, plasma exchange, and targeted biological agents) may help to manage symptoms, many patients still suffer from poor response, drug intolerance and disease relapse, highlighting a significant unmet clinical need. Telitacicept is the world's first approved innovative BLyS/APRIL dual-targeting fusion protein drug for MG. The pathology of MG is characterized by the autoantibodies produced by pathological B cells attacking the neuromuscular junction protein (such as acetylcholine receptor, muscle-specific tyrosine kinase). Telitacicept can block both BLyS and APRIL signaling pathways, effectively inhibiting abnormally activated B cells and reducing the production of pathogenic autoantibodies, which is expected to interfere with the source of disease progress of MG. Telitacicept was approved for marketing in China in May this year. Its phase III clinical trial boasts excellent results: after treatment with telitacicept for 24 weeks, improvement ≥ 3 points in myasthenia gravis-activities of daily living (MG-ADL) was observed in 98.1% of participants (12.0% in the placebo group) and improvements ≥ 5 points in quantitative myasthenia gravis (QMG) score was observed in 87% of participants (16.0% in the placebo group), with statistically significant difference in efficacy from the placebo group and manageable safety profile. The ODD granted by EMA represents the recognition of telitacicept's innovative mechanism of action and potential in treating MG. RemeGen is proceeding the global multi-center phase III clinical trial of telitacicept in patients with MG to bring the breakthrough treatment option to more patients globally. View original content to download multimedia: SOURCE RemeGen Co., Ltd Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
06-06-2025
- Health
- Yahoo
Canadian mom's fight for her son with 4 rare diseases: 'I'll keep pushing until someone listens'
Ashley Bergen noticed the blisters on her son William's wrist shortly after he was born. During her pregnancy, she developed preeclampsia, a serious health condition that causes high blood pressure and can cause organ damage, and delivered William via emergency C-section. When the blisters formed where William's hospital bracelet had been, a hospital resident at the Oakville, Ont. hospital immediately told Bergen to go to SickKids hospital in Toronto. William remained hospitalized for a week before being transferred to SickKids, where doctors diagnosed him with Epidermolysis Bullosa (EB), a group of rare hereditary skin diseases in which the body is unable to form the proteins needed to keep the skin strong. People with EB are sometimes referred to as "butterfly children" because they have extremely fragile skin — like a butterfly's wing — and can develop blisters and sores just by touch. It's estimated that there are approximately 300 to 500 people in Canada living with EB. This article is for informational purposes only and is not a substitute for professional medical advice, diagnosis or treatment. Contact a qualified medical professional before engaging in any physical activity, or making any changes to your diet, medication or lifestyle. 'They came back and told us that he had Epidermolysis Bullosa (EB). As a 19-year-old new mom, I didn't know what to expect, and then suddenly, I had this extra-fragile baby. I was afraid to pick him up. Babies scratch themselves and rub their eyes, so he had a lot of blisters and wounds. My learning curve was quick,' said Bergen, now 35, in an interview with Yahoo Canada. The Niagara region-based mom learned that children with EB have such delicate skin that they require special clothing, bedding and more. 'I knew the condition made him fragile but I refused to keep him in a bubble, adjusting only when necessary,' she said. 'For 14 years, aside from uncomfortable wounds, and strange stares and comments from strangers, he lived like any other kid." ... I didn't know what to expect, and then suddenly, I had this extra-fragile baby. I was afraid to pick him upAshley Bergen Everything started to change for Bergen's family in 2022. In April of that year, Bergen noticed that William was suddenly losing weight and muscle that made it difficult to perform simple tasks, like putting on his backpack or getting out of bed. 'Trying to get attention from the medical system was difficult because nobody wanted to listen,' she said. Bergen says she showed her doctors photos of her eldest son taken only a few months apart that showed a marked difference in his appearance. William, now 16, was referred to an orthopedic surgeon, who quickly escalated the case to a neuromuscular and neurometabolic disorder specialist in Hamilton, Ont.. And then came a wave of rare medical diagnoses. Within a year, William was diagnosed with scoliosis, a musculoskeletal condition, and Myasthenia Gravis (MG) an autoimmune disorder that causes fatigue and upper body weakness and impacts the muscles used to make facial expressions, swallow, chew, speak and breathe. WIlliam was also diagnosed with Avoidant/Restrictive Food Intake Disorder (ARFID), a lesser known eating disorder in which people restrict what they eat not because they're afraid of gaining weight, but because they have an aversion to the texture or smells of different foods. Although William had always faced challenges, by the time he entered grade 10, he was a different child. Bergen said she's seen first-hand the need to support medically-complex children after what she described as a "near-fatal" event at a hospital. William spent most of 2024 in hospital due to his deteriorating health. He was categorized as "failure to thrive" due to his low body weight and muscle weakness caused by MG. Bergen said William was so weak, he couldn't lift a cup of water to his face. Although he required BiPAP (bilevel positive airway pressure) while he slept, his skin condition made it too painful for him to wear a mask. Combined with his scoliosis impacting his chest cavity and ability to breathe and expel carbon dioxide, he faced tremendous challenges just to be able to breathe comfortably. Bergen said her experience with the hospital was frustrating. William had initially been denied access to its pediatric complex care program because, allegedly, his case was not "complex enough." Eventually, he was accepted into complex care, which provides treatment through a coordinated plan between healthcare providers across different specialties while involving patients and their families. Communication between parents and healthcare providers is supposed to be a key tenant of the program — but Bergen said this wasn't her experience. According to Bergen, things escalated when William was admitted to the Pediatric Intensive Care Unit after she left the hospital to tend to her younger son. 'His oxygen needs had skyrocketed. I dropped everything and rushed back. When I arrived, he was struggling with a full oxygen mask,' she said. The next day, Bergen said William's CO2 levels had risen to an alarming level. 'No one had called me. My son had been calling for me and no one had told me.' Bergen said the incident left William traumatized. 'The doctors wanted to test how he did on room air, but he was terrified. He cried every time they tried to take his mask off,' she explained. 'Even today, William does everything he can to avoid ever going through that again.' It has taken everything I have to fight and I'll keep pushing until someone listensAshley Bergen Bergen faced a new challenge when it was time for William to be discharged: accessing home care. Initially, William was deemed ineligible for home care. It was only through Bergen's relentless advocacy, along with an occupational therapist who documented every detail of William's condition, that they finally secured a personal support worker seven days a week. 'It has taken everything I have to fight and I'll keep pushing until someone listens," she said. Although William was approved for specialized at-home support through Special Services At Home (SSAH), Bergen said she was told there was no funding available. "The Special Services at Home (SSAH) program helps eligible families of children with a developmental or physical disability pay for services in, or outside the family home. For example, the family can hire someone to help their child learn new skills or improve existing abilities," said an SSAH spokesperson in a statement. "Our government has invested an additional amount of nearly $133 million into the SSAH program which helped to support over 36,400 children with developmental or physical disabilities receive support from the Special Services at Home program in 2023-24. "Our government is also supporting families with children with severe disabilities through our CARE tax credit, which provides up to $8,250 per child in tax relief for eligible childcare expenses." Yahoo Canada followed up with SSAH, however their spokesperson did not address claims that funding had run out in 2024. 'William is 16, and soon he'll be an adult. When that happens, the support he has as a child will go away. And that is an even bigger concern…" Bergen said. "It would be a challenge to raise two healthy kids in today's economy. It's an extremely large challenge with a medically complex child and a regular, happy two-year-old.' To make ends meet, Bergen works two roles at YMCA, while also pursuing higher education to expand her career options. 'The financial struggles have gone on for years, and it's only gotten worse as William's needs have increased,' she said. Recognizing this hardship, Bergen's colleague, Theresa Ganton, helped her start a GoFundMe page to help ease some of the financial burden. Bergen estimates she spends approximately $17,000 out of pocket each year to pay for things like medications and physiotherapy equipment. William requires entreal feeding equipment (he receives nutrients via feeding tube) and will require a motorized wheelchair in the future. The system helps in some in other ways, it just doesn't. Thanks to the GoFundMe, Bergen has been able to offset some of the costs to care for William at home. She was even able to access an adaptable tricycle to help William remain mobile and enjoy time outdoors with his brother. He was invited to participate in the DEBRA Canada Ride for EB, an annual bike ride to raise funds and awareness for children and families impacted by the rare condition. "The system helps in some ways,' she says, 'but in other ways, it just doesn't.' The severity of William's condition cannot be ignored but Bergen is sure that they will fight till the very end. 'I want my kid to prove everyone wrong. I want him to be the one that has stood up for not only him, but for other kids. I want him to be 40 and sitting at that table at the hospital telling his story about how he didn't give up.' In the face of challenges, Bergen and William find strength in each other. When someone recommended placing William in a specialized care home, Bergen dismissed the idea without hesitation. 'I'm not giving up on him. I will never give up on him,' she said. 'William is a unique teenager — not because of his medical condition, but because of his choices,' said Bergen. His joie de vivre shines through in his extraordinary wish for Make-A-Wish: while some kids may want to go to Disney, William wants to visit Mennonite colonies in Mexico. 'I want to give him the gift of travel.' "I want people to see children needing complex care as humans, not numbers — defined by who they are, not their condition," Bergen said.
Yahoo
06-06-2025
- Health
- Yahoo
Canadian mom's fight for her son with 4 rare diseases: 'I'll keep pushing until someone listens'
Ashley Bergen noticed the blisters on her son William's wrist shortly after he was born. During her pregnancy, she developed preeclampsia, a serious health condition that causes high blood pressure and can cause organ damage, and delivered William via emergency C-section. When the blisters formed where William's hospital bracelet had been, a hospital resident at the Oakville, Ont. hospital immediately told Bergen to go to SickKids hospital in Toronto. William remained hospitalized for a week before being transferred to SickKids, where doctors diagnosed him with Epidermolysis Bullosa (EB), a group of rare hereditary skin diseases in which the body is unable to form the proteins needed to keep the skin strong. People with EB are sometimes referred to as "butterfly children" because they have extremely fragile skin — like a butterfly's wing — and can develop blisters and sores just by touch. It's estimated that there are approximately 300 to 500 people in Canada living with EB. This article is for informational purposes only and is not a substitute for professional medical advice, diagnosis or treatment. Contact a qualified medical professional before engaging in any physical activity, or making any changes to your diet, medication or lifestyle. 'They came back and told us that he had Epidermolysis Bullosa (EB). As a 19-year-old new mom, I didn't know what to expect, and then suddenly, I had this extra-fragile baby. I was afraid to pick him up. Babies scratch themselves and rub their eyes, so he had a lot of blisters and wounds. My learning curve was quick,' said Bergen, now 35, in an interview with Yahoo Canada. The Niagara region-based mom learned that children with EB have such delicate skin that they require special clothing, bedding and more. 'I knew the condition made him fragile but I refused to keep him in a bubble, adjusting only when necessary,' she said. 'For 14 years, aside from uncomfortable wounds, and strange stares and comments from strangers, he lived like any other kid." ... I didn't know what to expect, and then suddenly, I had this extra-fragile baby. I was afraid to pick him upAshley Bergen Everything started to change for Bergen's family in 2022. In April of that year, Bergen noticed that William was suddenly losing weight and muscle that made it difficult to perform simple tasks, like putting on his backpack or getting out of bed. 'Trying to get attention from the medical system was difficult because nobody wanted to listen,' she said. Bergen says she showed her doctors photos of her eldest son taken only a few months apart that showed a marked difference in his appearance. William, now 16, was referred to an orthopedic surgeon, who quickly escalated the case to a neuromuscular and neurometabolic disorder specialist in Hamilton, Ont.. And then came a wave of rare medical diagnoses. Within a year, William was diagnosed with scoliosis, a musculoskeletal condition, and Myasthenia Gravis (MG) an autoimmune disorder that causes fatigue and upper body weakness and impacts the muscles used to make facial expressions, swallow, chew, speak and breathe. WIlliam was also diagnosed with Avoidant/Restrictive Food Intake Disorder (ARFID), a lesser known eating disorder in which people restrict what they eat not because they're afraid of gaining weight, but because they have an aversion to the texture or smells of different foods. Although William had always faced challenges, by the time he entered grade 10, he was a different child. Bergen said she's seen first-hand the need to support medically-complex children after what she described as a "near-fatal" event at a hospital. William spent most of 2024 in hospital due to his deteriorating health. He was categorized as "failure to thrive" due to his low body weight and muscle weakness caused by MG. Bergen said William was so weak, he couldn't lift a cup of water to his face. Although he required BiPAP (bilevel positive airway pressure) while he slept, his skin condition made it too painful for him to wear a mask. Combined with his scoliosis impacting his chest cavity and ability to breathe and expel carbon dioxide, he faced tremendous challenges just to be able to breathe comfortably. Bergen said her experience with the hospital was frustrating. William had initially been denied access to its pediatric complex care program because, allegedly, his case was not "complex enough." Eventually, he was accepted into complex care, which provides treatment through a coordinated plan between healthcare providers across different specialties while involving patients and their families. Communication between parents and healthcare providers is supposed to be a key tenant of the program — but Bergen said this wasn't her experience. According to Bergen, things escalated when William was admitted to the Pediatric Intensive Care Unit after she left the hospital to tend to her younger son. 'His oxygen needs had skyrocketed. I dropped everything and rushed back. When I arrived, he was struggling with a full oxygen mask,' she said. The next day, Bergen said William's CO2 levels had risen to an alarming level. 'No one had called me. My son had been calling for me and no one had told me.' Bergen said the incident left William traumatized. 'The doctors wanted to test how he did on room air, but he was terrified. He cried every time they tried to take his mask off,' she explained. 'Even today, William does everything he can to avoid ever going through that again.' It has taken everything I have to fight and I'll keep pushing until someone listensAshley Bergen Bergen faced a new challenge when it was time for William to be discharged: accessing home care. Initially, William was deemed ineligible for home care. It was only through Bergen's relentless advocacy, along with an occupational therapist who documented every detail of William's condition, that they finally secured a personal support worker seven days a week. 'It has taken everything I have to fight and I'll keep pushing until someone listens," she said. Although William was approved for specialized at-home support through Special Services At Home (SSAH), Bergen said she was told there was no funding available. "The Special Services at Home (SSAH) program helps eligible families of children with a developmental or physical disability pay for services in, or outside the family home. For example, the family can hire someone to help their child learn new skills or improve existing abilities," said an SSAH spokesperson in a statement. "Our government has invested an additional amount of nearly $133 million into the SSAH program which helped to support over 36,400 children with developmental or physical disabilities receive support from the Special Services at Home program in 2023-24. "Our government is also supporting families with children with severe disabilities through our CARE tax credit, which provides up to $8,250 per child in tax relief for eligible childcare expenses." Yahoo Canada followed up with SSAH, however their spokesperson did not address claims that funding had run out in 2024. 'William is 16, and soon he'll be an adult. When that happens, the support he has as a child will go away. And that is an even bigger concern…" Bergen said. "It would be a challenge to raise two healthy kids in today's economy. It's an extremely large challenge with a medically complex child and a regular, happy two-year-old.' To make ends meet, Bergen works two roles at YMCA, while also pursuing higher education to expand her career options. 'The financial struggles have gone on for years, and it's only gotten worse as William's needs have increased,' she said. Recognizing this hardship, Bergen's colleague, Theresa Ganton, helped her start a GoFundMe page to help ease some of the financial burden. Bergen estimates she spends approximately $17,000 out of pocket each year to pay for things like medications and physiotherapy equipment. William requires entreal feeding equipment (he receives nutrients via feeding tube) and will require a motorized wheelchair in the future. The system helps in some in other ways, it just doesn't. Thanks to the GoFundMe, Bergen has been able to offset some of the costs to care for William at home. She was even able to access an adaptable tricycle to help William remain mobile and enjoy time outdoors with his brother. He was invited to participate in the DEBRA Canada Ride for EB, an annual bike ride to raise funds and awareness for children and families impacted by the rare condition. "The system helps in some ways,' she says, 'but in other ways, it just doesn't.' The severity of William's condition cannot be ignored but Bergen is sure that they will fight till the very end. 'I want my kid to prove everyone wrong. I want him to be the one that has stood up for not only him, but for other kids. I want him to be 40 and sitting at that table at the hospital telling his story about how he didn't give up.' In the face of challenges, Bergen and William find strength in each other. When someone recommended placing William in a specialized care home, Bergen dismissed the idea without hesitation. 'I'm not giving up on him. I will never give up on him,' she said. 'William is a unique teenager — not because of his medical condition, but because of his choices,' said Bergen. His joie de vivre shines through in his extraordinary wish for Make-A-Wish: while some kids may want to go to Disney, William wants to visit Mennonite colonies in Mexico. 'I want to give him the gift of travel.' "I want people to see children needing complex care as humans, not numbers — defined by who they are, not their condition," Bergen said.
Business Upturn
02-06-2025
- Business
- Business Upturn
ImmunAbs Announces FDA Phase 2 IND Approval of IM-101, a Novel Complement C5 Inhibitor, for Treatment of Myasthenia Gravis
By Business Wire Published on June 2, 2025, 17:07 IST Seoul, South Korea: ImmunAbs Inc., a clinical-stage biotech specializing in developing antibody therapeutics, today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application to initiate a Phase 2 clinical trial evaluating the safety and efficacy of IM-101, a novel complement C5 inhibitor, for the treatment of Myasthenia Gravis. The forthcoming multicenter, randomized, double-blind, placebo-controlled study will enroll up to 90 patients to evaluate the effectiveness and safety of monthly IM-101 dosing in relieving complement-induced symptoms in MG. 'This IND approval is pivotal for us as it brings us one step closer to delivering a transformative therapy for patients with autoimmune disorders', said Dr. Dongjo Kim, the CEO of ImmunAbs, 'we believe IM-101 has the potential to deliver deeper therapeutic responses and more durable remissions in patients who have failed to achieve sustained remission with current approved treatment.' About Myasthenia Gravis Myasthenia Gravis (MG) is a rare neurological disorder that severely impairs muscle movement and can lead to life-threatening complications. While existing C5 inhibitors have contributed to improved remission rates, many patients still experience residual disease activity due to incomplete complement inhibition. In addition to autoantibody-mediated activation of the classical complement pathway, MG pathogenesis is also significantly influenced by activation of the alternative pathway. ImmunAbs believes that comprehensive inhibition of both the classical and alternative pathways is essential for minimizing residual complement activity and enabling revolutionary treatments for MG. About IM-101 IM-101 is a humanized monoclonal antibody targeting complement C5 with high affinity. In the recently concluded Phase 1 clinical trial, IM-101 demonstrated an excellent safety profile and was well tolerated at all tested doses, with no dose-limiting toxicity or severe adverse events observed. The clinical results showcasing IM-101's outstanding ability to reduce serum free C5 concentrations in healthy volunteers provide strong evidence of its superior efficacy in complement inhibition. About ImmunAbs ImmunAbs is a clinical-stage biotechnology company established in 2017. Committed to delivering transformative treatments globally, the company aims to develop novel antibody therapeutics, particularly concentrating its efforts on the lead program targeting severe autoimmune diseases. Supported by equity funding and national grants from KDDF, which covered development from non-clinical through Phase 1 clinical studies, ImmunAbs is now seeking global partnerships to accelerate the advancement of IM-101 towards market availability. View source version on Disclaimer: The above press release comes to you under an arrangement with Business Wire. Business Upturn takes no editorial responsibility for the same. Business Wire is an American company that disseminates full-text press releases from thousands of companies and organizations worldwide to news media, financial markets, disclosure systems, investors, information web sites, databases, bloggers, social networks and other audiences.
