Latest news with #musculardystrophy
Yahoo
9 hours ago
- Health
- Yahoo
Gymgoers to lift the weight of a jumbo jet for little boy with rare condition
GYMGOERS are set to lift the weight of a jumbo jet to help raise money for a little boy with a severe genetic condition. Colin Morgan, 67, is leading the charge at the Flow Gym, in West Mersea, after being contacted by Jacob Ackerman's parents Lydia and her husband Harry. Jacob was diagnosed with muscular dystrophy at just two years old. Strength - Jacob Ackerman was diagnosed with muscular dystrophy aged two (Image: Submitted)Muscular dystrophy is a severe genetic condition which gradually causes the muscles to weaken and can become life-threatening if it affects the heart. There is no cure for muscular dystrophy, but treatment can help with many of the symptoms. Jacob's Jumbo Lift will see a team of 12 men and women from the gym attempt to lift the equivalent weight of a fully laden Boeing 747-400 Jumbo Jet in aid of the charity Muscular Dystrophy UK. Organiser - Challenge organiser Colin Morgan (Image: Submitted)Mr Morgan said the team named the The Dumbbell Dozen have been 'putting in some serious training and are in great shape'. He said: 'We are absolutely confident of hitting our target total weight of 420 tonnes and indeed expect to add at least a couple of London buses, a Lear Jet and a family of African elephants to the final total." Mr Morgan said fundraising has reached more than £3,500. A total of £50 would fund one hour of high-quality research, £100 raised will fund a day of helpline operation, and £200 will fund an information day, bringing experts and the MD community together. Fundraiser - The month-long fundraiser will raise money for Muscular Dystrophy UK (Image: Submitted) Team - The Dumbbell Dozen will be wearing branded t-shirts as part of the challenge, raising awareness (Image: Submitted) There will also be high-profile visitors to the challenge with Sir Bernard Jenkin, MP for Harwich and North Essex, visiting the challenge in action on July 11. The mayors of West Mersea and Colchester will also be in attendance, along with the national chief executive of Muscular Dystrophy UK, Andy Fletcher. For more updates including progress on the challenge visit
Yahoo
2 days ago
- Business
- Yahoo
BridgeBio to Host Limb-girdle Muscular Dystrophy Type 2I/R9 Investor Webinar on Friday, July 11th at 8:00 am ET
PALO ALTO, Calif., June 26, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a new type of biopharmaceutical company focused on genetic diseases, today announced the Company will host an investor webinar on Friday, July 11, 2025 at 8:00 am ET with Matthew Wicklund, M.D., FAAN, Professor of Neurology and Vice Chair for Research, Department of Neurology at the University of Texas (UT) Health Science Center San Antonio. Dr. Wicklund will provide an overview of limb-girdle muscular dystrophy (LGMD), specifically focusing on the disease burden, standard of care and unmet needs for those with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) (FKRP-related). He serves as director for the UT Health San Antonio MDA Multidisciplinary Care Center and is a member of the Limb-girdle Muscular Dystrophy Clinical Research Network and the Neuromuscular Study Group. Dr. Wicklund has participated in over 35 multi-center clinical trials in the areas of muscular dystrophy, ALS, and myasthenia gravis and has over 200 published articles, chapters and abstracts. In addition to Dr. Wicklund, executive members of the LGMD2I/R9 program will review the progress of BBP-418 to date and discuss expectations for the Phase 3 interim analysis results expected in the second half of 2025. To access the live webcast of BridgeBio's investor webinar, please visit the 'Events & Presentations' page within the Investors section of the BridgeBio website at A replay of the webcast will be available on the BridgeBio website for 30 days following the event. About BridgeBio Pharma, Pharma, Inc. (BridgeBio) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit and follow us on LinkedIn, Twitter, Facebook, and YouTube. BridgeBio Media Contact:Bubba Murarka, Executive Vice Presidentcontact@ (650)-789-8220 BridgeBio Investor Contact:Chinmay Shukla, VP Strategic Financeir@ while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
The Sun
5 days ago
- Entertainment
- The Sun
Brit ‘making £10,000 a MONTH' from helping Fortnite players online and says he even quit sixth form ‘after a day'
ONE YOUNG Brit says he makes a staggering £10,000 a month from a popular video game. The gaming enthusiast quit sixth form 'after a day' in order to pursue his highly-lucrative career - where he manages Fortnite players. 3 3 Ross McLaren, 22, originally wanted to be a professional Fortnite player and spent hours at home practising. Ross has muscular dystrophy - a disease which causes muscles in the body to weaken over time - but he thinks that was an 'advantage' in his career. He said: 'Having muscular dystrophy was somewhat of an advantage for me as it meant I spent a lot more time on a computer when I was younger, as I couldn't go out and play sports like other kids.' His dreams of becoming a 'pro Fortnite player' were dashed when he realised he 'wasn't good enough' to match up to some of the best players in the world. However, Ross knew that he could put his talents to good use. At the age of 17, while still living with his parents Lesley and Craig, he decided to manage other players. His career quickly took off and he began to make an eye-watering sum of money. Ross said: 'By summer 2021, I was making £10,000 per month, and I told my parents I didn't want to sit my Advanced Highers.' His parents encouraged him to go back to school, but Ross quit after just one day. Ross said that he got his big break when he worked with Bugha, a streamer who only had 20,000 followers at the time. Gamer granny 'hooked' on Fortnite at 76 - and says it was 'love at first sight' after amassing 20k followers on Twitch When Bugha won the Fortnite World Cup in 2018 and won $3 million, his subscriber count rocketed to one million. Soon, Ross became inundated with business opportunities with players begging him to edit videos of them playing the game. After leaving George Heriot's School, the tech expert started running KSI's gaming channel; - which has over 22 million subscribers. As his star continues to rise, Ross is eyeing a move to Dallas, Texas, where many of the biggest Fortnite streamers are based. The news comes after the makers of an online game with 40 million players will be removed from two consoles. Black Desert Online has long been one of the most popular games in the industry, but will soon no longer be available on PS4 and XBox One. Players will have to buy a Xbox Series X/S and PlayStation 5 to play the game from June 26. The game's developers, Pearl Abyss, said: "We understand that this news may be disappointing for our longtime Adventurers on these platforms. "Please know that this decision was made after careful consideration, with the goal of ensuring the future growth and evolution of Black Desert Console. "We remain fully committed to delivering you the best possible adventures in Black Desert." 3
Yahoo
15-06-2025
- Health
- Yahoo
[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients
After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been re-assessed, following two cases of fatal acute liver failure Effective immediately, dosing of non-ambulatory patients, irrespective of age, is paused in the clinical setting; dosing of non-ambulatory patients is discontinued in the commercial setting Roche is working closely with relevant health authorities, investigators and prescribing physicians to ensure they are informed and patient care is being appropriately modified The benefit-risk of Elevidys treatment in ambulatory Duchenne patients remains positive and treatment guidance is unchanged Basel, 15 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. In the commercial setting, non-ambulatory patients should no longer receive Elevidys. In the clinical trial setting, enrolment and dosing of non-ambulatory patients will be immediately paused until additional risk mitigation measures (e.g. immune modulatory treatment) are implemented in the study protocol. Health authorities, investigators and physicians are being informed so that patient care can be quickly adjusted. This decision follows careful assessment of two cases in non-ambulatory patients of fatal acute liver failure (ALF), an identified risk of Elevidys and other AAV-mediated gene therapies, which led to a reassessment of the benefit-risk profile as unfavourable for patients with DMD who are non-ambulatory. The new dosing restrictions do not impact the treatment of ambulatory DMD patients of any age, and the benefit-risk ratio remains positive in the ambulatory patient population. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect.' DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. The two fatal ALF cases occurred in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date. Following the first case of fatal ALF, European regulators requested that Roche and Sarepta put temporary clinical holds on Elevidys studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). The temporary clinical holds are still in effect. Outside of Europe, dosing will be paused, effective immediately, for the ENVISION trial. The dosing restrictions will also go into effect for future dosing of commercial non-ambulatory patients. Elevidys has been approved by regulatory authorities in eight Roche territories for the treatment of DMD including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the UAE. In 2019, Roche entered into a global collaboration agreement with Sarepta Therapeutics, Inc. to commercialise Elevidys in territories outside the U.S. Roche and Sarepta jointly manage the clinical studies for Elevidys. Roche is the sponsor of the ENVOL study; Sarepta is the sponsor for all other studies. Overview of the Elevidys clinical development programme Studies in non-ambulatory people with DMD Ongoing ENVISION (Study 303, NCT05881408), a global Phase III study investigating the safety and efficacy of Elevidys in participants who are ambulatory (aged 8 to <18 years old) and non-ambulatory (no age limitation). This study is already on temporary clinical hold in Europe. Outside of Europe, recruitment will be paused. ENDEAVOR (Study 103, NCT04626674), a two-part, open-label, Phase Ib study assessing Elevidys micro-dystrophin protein expression and safety of Elevidys in seven cohorts of boys with Duchenne, across different ages, mutations and stages of disease progression. No longer recruiting; long term follow up ongoing. Studies in ambulatory people with DMD Study 101 (NCT03375164), a Phase I/II study evaluating the safety of Elevidys in four ambulatory participants aged 4 to <8 years old with Duchenne. The study is complete. Study 102 (NCT03769116), a Phase II clinical trial evaluating the safety and efficacy of Elevidys in patients with Duchenne aged 4 to <8 years. The study is complete. Study 104 (NCT06241950), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys in association with imlifidase in individuals aged 4 to 9 years with pre-existing antibodies to recombinant adeno-associated virus serotype, rAAVrh74. The study is on temporary clinical hold in Europe. HORIZON (Study 105, NCT06597656), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys following plasmapheresis in individuals aged 4 to 8 years with pre-existing antibodies to adeno-associated virus serotype, AAVrh74. The study is recruiting ambulatory patients. EMBARK (Study 301, NCT05096221), a multinational, Phase III, randomised, double-blind, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged 4 to 7 years. The study duration is two years. The study is complete. ENVOL (Study 302, NCT06128564), a Phase II study evaluating the safety of Elevidys and expression of Elevidys micro-dystrophin protein in young children, including babies and newborns. The study is on temporary clinical hold in Europe and the UK. EXPEDITION (Study 305, NCT05967351), a Phase III long-term five-year follow-up study evaluating the safety and efficacy of Elevidys in those who have received Elevidys in a previous clinical study. EXPEDITION is enrolling by invitation. About Elevidys™ (delandistrogene moxeparvovec) Elevidys is a one-time treatment administered through a single intravenous dose and the first and only approved gene therapy for Duchenne. It is designed to target the underlying cause of Duchenne by delivering new instructions to cells to produce Elevidys-dystrophin in skeletal, respiratory and cardiac muscles. Elevidys aims to slow the progression of Duchenne by delaying the need for a wheelchair, protecting the heart from damage and a person's ability to breathe without a respirator for as long as possible. Elevidys uses adeno-associated virus (AAV) vector technology and consists of three parts: a transgene, promoter and vector. Its unique construct optimises delivery to all muscle types, including those of interest for Duchenne treatment. A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is ongoing. To date, more than 900 individuals with Duchenne have been treated with Elevidys through Roche's clinical development program and in real-world settings. Elevidys has already been approved for the treatment of DMD by 10 regulatory authorities around the world, including the US and Japan. Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics. About Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. Average life expectancy is only 28 years. The physical, emotional and financial impact of Duchenne on those affected, their families and caregivers, is profound. Duchenne is an X-linked, rare neuromuscular disease caused by pathogenic variants (mutations) in the DMD gene that disrupt the production of functional dystrophin protein, leading to progressive and irreversible muscle weakness, diminished quality of life and premature death. Dystrophin strengthens and protects muscles and without it, normal activity causes excessive damage to muscle cells as they are more sensitive to injury. Over time, muscle tissue is replaced with scar tissue and fat, causing muscles to weaken. Although Duchenne progresses differently in each individual, its devastating trajectory is well established. Those with Duchenne will eventually lose the ability to use and move their limbs, to breathe on their own and are susceptible to respiratory infections. Muscle damage to the heart causes cardiomyopathy, including rhythm abnormalities and heart failure. Early diagnosis is important for timely intervention to prolong muscle function and preserve quality of life. There is a critical need for disease-modifying treatments that address the underlying cause of DMD before irreversible muscle loss occurs. About Roche in Neuroscience Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer's disease, Huntington's disease, Parkinson's disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today. About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit All trademarks used or mentioned in this release are protected by law. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Nina Mählitz Phone: +41 79 327 54 74 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Dr Bruno Eschli Phone: +41 61 68-75284 e-mail: Dr Sabine Borngräber Phone: +41 61 68-88027 e-mail: Dr Birgit Masjost Phone: +41 61 68-84814 e-mail: Investor Relations North America Loren Kalm Phone: +1 650 225 3217 e-mail: Attachment Ad Hoc Media Investor Release Update on Elevidys EnglishError 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
15-06-2025
- Health
- Yahoo
[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients
After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been re-assessed, following two cases of fatal acute liver failure Effective immediately, dosing of non-ambulatory patients, irrespective of age, is paused in the clinical setting; dosing of non-ambulatory patients is discontinued in the commercial setting Roche is working closely with relevant health authorities, investigators and prescribing physicians to ensure they are informed and patient care is being appropriately modified The benefit-risk of Elevidys treatment in ambulatory Duchenne patients remains positive and treatment guidance is unchanged Basel, 15 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. In the commercial setting, non-ambulatory patients should no longer receive Elevidys. In the clinical trial setting, enrolment and dosing of non-ambulatory patients will be immediately paused until additional risk mitigation measures (e.g. immune modulatory treatment) are implemented in the study protocol. Health authorities, investigators and physicians are being informed so that patient care can be quickly adjusted. This decision follows careful assessment of two cases in non-ambulatory patients of fatal acute liver failure (ALF), an identified risk of Elevidys and other AAV-mediated gene therapies, which led to a reassessment of the benefit-risk profile as unfavourable for patients with DMD who are non-ambulatory. The new dosing restrictions do not impact the treatment of ambulatory DMD patients of any age, and the benefit-risk ratio remains positive in the ambulatory patient population. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect.' DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. The two fatal ALF cases occurred in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date. Following the first case of fatal ALF, European regulators requested that Roche and Sarepta put temporary clinical holds on Elevidys studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). The temporary clinical holds are still in effect. Outside of Europe, dosing will be paused, effective immediately, for the ENVISION trial. The dosing restrictions will also go into effect for future dosing of commercial non-ambulatory patients. Elevidys has been approved by regulatory authorities in eight Roche territories for the treatment of DMD including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the UAE. In 2019, Roche entered into a global collaboration agreement with Sarepta Therapeutics, Inc. to commercialise Elevidys in territories outside the U.S. Roche and Sarepta jointly manage the clinical studies for Elevidys. Roche is the sponsor of the ENVOL study; Sarepta is the sponsor for all other studies. Overview of the Elevidys clinical development programme Studies in non-ambulatory people with DMD Ongoing ENVISION (Study 303, NCT05881408), a global Phase III study investigating the safety and efficacy of Elevidys in participants who are ambulatory (aged 8 to <18 years old) and non-ambulatory (no age limitation). This study is already on temporary clinical hold in Europe. Outside of Europe, recruitment will be paused. ENDEAVOR (Study 103, NCT04626674), a two-part, open-label, Phase Ib study assessing Elevidys micro-dystrophin protein expression and safety of Elevidys in seven cohorts of boys with Duchenne, across different ages, mutations and stages of disease progression. No longer recruiting; long term follow up ongoing. Studies in ambulatory people with DMD Study 101 (NCT03375164), a Phase I/II study evaluating the safety of Elevidys in four ambulatory participants aged 4 to <8 years old with Duchenne. The study is complete. Study 102 (NCT03769116), a Phase II clinical trial evaluating the safety and efficacy of Elevidys in patients with Duchenne aged 4 to <8 years. The study is complete. Study 104 (NCT06241950), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys in association with imlifidase in individuals aged 4 to 9 years with pre-existing antibodies to recombinant adeno-associated virus serotype, rAAVrh74. The study is on temporary clinical hold in Europe. HORIZON (Study 105, NCT06597656), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys following plasmapheresis in individuals aged 4 to 8 years with pre-existing antibodies to adeno-associated virus serotype, AAVrh74. The study is recruiting ambulatory patients. EMBARK (Study 301, NCT05096221), a multinational, Phase III, randomised, double-blind, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged 4 to 7 years. The study duration is two years. The study is complete. ENVOL (Study 302, NCT06128564), a Phase II study evaluating the safety of Elevidys and expression of Elevidys micro-dystrophin protein in young children, including babies and newborns. The study is on temporary clinical hold in Europe and the UK. EXPEDITION (Study 305, NCT05967351), a Phase III long-term five-year follow-up study evaluating the safety and efficacy of Elevidys in those who have received Elevidys in a previous clinical study. EXPEDITION is enrolling by invitation. About Elevidys™ (delandistrogene moxeparvovec) Elevidys is a one-time treatment administered through a single intravenous dose and the first and only approved gene therapy for Duchenne. It is designed to target the underlying cause of Duchenne by delivering new instructions to cells to produce Elevidys-dystrophin in skeletal, respiratory and cardiac muscles. Elevidys aims to slow the progression of Duchenne by delaying the need for a wheelchair, protecting the heart from damage and a person's ability to breathe without a respirator for as long as possible. Elevidys uses adeno-associated virus (AAV) vector technology and consists of three parts: a transgene, promoter and vector. Its unique construct optimises delivery to all muscle types, including those of interest for Duchenne treatment. A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is ongoing. To date, more than 900 individuals with Duchenne have been treated with Elevidys through Roche's clinical development program and in real-world settings. Elevidys has already been approved for the treatment of DMD by 10 regulatory authorities around the world, including the US and Japan. Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics. About Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. Average life expectancy is only 28 years. The physical, emotional and financial impact of Duchenne on those affected, their families and caregivers, is profound. Duchenne is an X-linked, rare neuromuscular disease caused by pathogenic variants (mutations) in the DMD gene that disrupt the production of functional dystrophin protein, leading to progressive and irreversible muscle weakness, diminished quality of life and premature death. Dystrophin strengthens and protects muscles and without it, normal activity causes excessive damage to muscle cells as they are more sensitive to injury. Over time, muscle tissue is replaced with scar tissue and fat, causing muscles to weaken. Although Duchenne progresses differently in each individual, its devastating trajectory is well established. Those with Duchenne will eventually lose the ability to use and move their limbs, to breathe on their own and are susceptible to respiratory infections. Muscle damage to the heart causes cardiomyopathy, including rhythm abnormalities and heart failure. Early diagnosis is important for timely intervention to prolong muscle function and preserve quality of life. There is a critical need for disease-modifying treatments that address the underlying cause of DMD before irreversible muscle loss occurs. About Roche in Neuroscience Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer's disease, Huntington's disease, Parkinson's disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today. About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit All trademarks used or mentioned in this release are protected by law. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Nina Mählitz Phone: +41 79 327 54 74 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Dr Bruno Eschli Phone: +41 61 68-75284 e-mail: Dr Sabine Borngräber Phone: +41 61 68-88027 e-mail: Dr Birgit Masjost Phone: +41 61 68-84814 e-mail: Investor Relations North America Loren Kalm Phone: +1 650 225 3217 e-mail: Attachment Ad Hoc Media Investor Release Update on Elevidys EnglishError 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
