Latest news with #spinalmuscularatrophy
Globe and Mail
a day ago
- Business
- Globe and Mail
Scholar Rock to Host Conference Call to Discuss Second Quarter 2025 Financial Results and Provide Business Update on August 6, 2025
Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with spinal muscular atrophy (SMA) and other severe and debilitating neuromuscular diseases, today announced that management will host a conference call to discuss its second quarter 2025 financial results and provide a business update on Wednesday, August 6, 2025, at 8:00am ET. To access the live conference call, participants may register here. The live audio webcast of the call will be available under 'Events and Presentations' in the Investor Relations section of the Scholar Rock website at To participate via telephone, please join by dialing 800-715-9871 (domestic) or 646-307-1963 (international) and referencing the conference ID 3205013. An archived replay of the webcast will be available on the Company's website for approximately 90 days. About Scholar Rock Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily, the company is named for the visual resemblance of a scholar rock to protein structures. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role. This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at and follow @ScholarRock on LinkedIn. Scholar Rock ® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X (formerly known as Twitter) or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
Sky News
2 days ago
- Health
- Sky News
Mother of baby with spinal muscular atrophy wants all newborns to be tested for it
The mother of baby boy, who had 22 medical appointments with nine different doctors before being diagnosed with a rare genetic condition, wants all newborns to be tested for it. Chester was eight months old when his parents were finally told the condition that meant he couldn't swallow or move his legs and left him constantly distressed was spinal muscular atrophy, or SMA. But it's a delay that will have a lifelong impact. "He still can't hold his head up," said his mum, Kasey Mumford. "If he'd been diagnosed by the heel prick test at like three or five days old, he could have started medication straight away, because there's a treatment for it, there are three different types of treatment, but there's no cure. He will never not have this." Chester is nearly one now, but needs a ventilator at night to breathe. But SMA isn't one of the 10 rare illnesses that newborn babies are currently tested for in the UK, unlike in the US and much of Europe. Dr Vasantha Gowda, a consultant paediatric neurologist at London's St Thomas' Hospital, says clinicians from other countries are "all shocked that we don't have newborn screening for SMA". "If we treat these children early, there is a possibility that… they would walk, run, climb, play. They would not need any support for breathing. They would need no support for feeding," she said. "We know there's a potential to deliver this if the condition is picked up at the earliest possible stage." SMA causes irreversible loss of motor neurons, which affects muscle function. Nine out of 10 will either die before they're two or rely on a ventilator for life. And when 2,000 parents were surveyed recently, 90% said they'd want to know as soon as possible if their child had SMA. Four in every five wanted screening implemented immediately, and the same proportion considered the £5 cost per test good value. The lack of testing for the condition is, according to the chief executive of the charity SMA UK, "unethical". "The new 10-year NHS plan has highlighted that prevention is a key pillar," said Giles Lomax. "Newborn screening for SMA is essentially a very quick and easy win to align to that pillar. "We've got the treatments there, we've got pathways, we have the expertise, we could make prevention for SMA a thing right now." New therapies have been developed since SMA was last considered for inclusion, and in 2023, the UK National Screening Committee agreed to review the decision. A large-scale study is now under way and the different governments across the UK have said they'll be guidedby the advisory body's recommendations. But Chester's mum is clear that it must be included as "every single second counts" when it comes to receiving treatment. Instead, for Kasey and her partner, Dylan, there will always be that uncertainty about what might have been had their boy been diagnosed sooner.
The National
4 days ago
- Health
- The National
Syrian girl receives life-saving treatment as Dubai Ruler covers Dh7 million cost
A young Syrian girl diagnosed with spinal muscular atrophy received Dh7 million gene therapy paid for by Sheikh Mohammed bin Rashid, Vice President and Ruler of Dubai, on Friday at the emirate's Al Jalila Children's Speciality Hospital. Holding a small giraffe toy, and trying to smile, Yaqeen Kankar, sat in the treatment room on Friday morning waiting for the arrival of doctors and her medicine. Two weeks ago, the family made a video appeal on social media for help to save the two-year-old. The office of Sheikh Mohammed called the family saying the Ruler of Dubai would cover the Dh7 million ($1.9 million) cost of medical treatment. Yaqeen's uncle, Ibrahim Abdulaziz Faroj, told The National the family was relieved to know she could now have a normal life. 'We didn't sleep for three days after receiving the happy news that Sheikh Mohammed would cover the cost,' Mr Faroj, 23, said. 'It's a mix of feelings between joy, anticipation and anxiety. We want her to grow normally like any other child. Thanks to Dubai, the dream becomes true.' The uncle had posted a video holding Yaqeen, explaining her condition and asking for help with the cost of her treatment, which is available at only a few centres in the world. 'We know that she has no time. We didn't have other way than to plea for help as we couldn't afford the cost,' Mr Faroj said. Eight months before moving to Dubai, doctors in Syrian diagnosed Yaqeen with spinal muscular atrophy (SMA), a genetic disorder that weakens and wastes muscles. Her parents left no stone unturned seeking help. 'I was posting about her for several months until we received the happy call,' Mr Faroj said. 'It was the best gift ever. We all cried with joy when we heard the news. This action is not strange for the UAE and its rulers who always help people in desperate need.' Doctors gave Yaqeen only months to live without the expensive treatment. Dr Haitham Elbashir, paediatric neurorehabilitation consultant at Al Jalila Children's Hospital, told The National: 'We examined her a week ago and did all the laboratory tests. I'm very happy to say that the medicine is going to be delivered to her today.' Yaqeen underwent the one-hour procedure on Friday morning and will stay in the hospital for 24 hours. Then she can go with her parents to their home in Sharjah. Doctors said they were optimistic it would be successful, but it could take up to three months to see the final results. 'She will need to continue with us for three months because that's the treatment period where we need to follow her regularly,' said Dr Elbashir. Yaqeen was diagnosed with SMA type 2 when she was only six months old. 'Her condition is stable, but she's got some weakness and hopefully the treatment will help to get her better. I'm very optimistic,' said Dr Elbashir. SMA affects the motor neurons (nerve cells that transmit impulses to the muscles) and weakens the limbs. It makes walking difficult or impossible and creates problems with swallowing, as well as breathing. The treatment offered by Al Jalila Children's Hospital, called Zolgensma, is given once and is a form of gene therapy. It was approved by the US Food and Drug Administration four years ago and has been described as the most expensive drug in the world. The hospital has completed more than 100 treatments for patients with SMA, said Dr Mohamed Al Awadhi, executive director of the Dubai Health Women and Children's Campus.
The National
03-07-2025
- Health
- The National
‘A father to all children': Sheikh Mohammed funds life-saving treatment for Syrian girl
Sheikh Mohammed bin Rashid, Vice President and Ruler of Dubai, will cover Dh7 million ($1.9 million) in medical treatment for a young girl suffering from spinal muscular atrophy. The announcement came after Yaqeen's uncle posted a video on social media appealing for help to save her life. In the video, he said the Syrian girl, who will soon turn two, suffers from a degenerative and debilitating disease. Doctors had diagnosed her with spinal muscle atrophy (SMA) – a genetic disorder that weakens and wastes muscles away, and her uncle asked for help in the cost of her treatment, which is available in only a few countries, including Al Jalila Hospital in Dubai. Yaqeen's father, Ibrahim Kankar, 27, told The National he couldn't believe it when someone from the office of Sheikh Mohammed informed him that the Ruler of Dubai would cover the costs in full. 'Sheikh Mohammed bin Rashid is the father of all children. This action is not strange from the UAE and its rulers,' Mr Kankar said. 'I couldn't believe that three days after posting the video, with the help of the UAE's Charity International Organisation, my daughter will survive and live a normal childhood. You don't know how that feels.' Critical need Mr Kankar, who lives in Sharjah, said doctors gave Yaqeen only months to live if she could not undergo the expensive treatment. 'Her health condition is very critical and she needs urgent treatment which we can't afford,' he added. 'Me and her mother are thrilled and full of joy knowing she will now get it. These unforgotten moments will last long in our memory.' Yaqeen is Mr Kankar's only child. 'She has symptoms that we couldn't understand like not being able to interact with her surroundings. After many failed attempts to diagnose her disease, we came to the UAE where we found care and mercy,' he said. Dr Khaled Abdul Wahab Al Khaja, secretary general of the International Charity Organisation, praised the generous gesture. 'Sheikh Mohammed represents a model for unlimited humanitarian work,' Mr Al Khaja said. The organisation sponsored the girl's case by posting a plea on their website and social media. 'Her family has a poor income and it was impossible for them to pay the cost of treatment,' Mr Al Khaja added. "Sheikh Mohammed is the supporter for any person in need in the UAE." Looking to the future Dubai Health's Al Jalila Children Hospital welcomed Yaqeen on Thursday and examined her with the necessary tests. "The medical team has commenced essential diagnostic procedures, including genetic and antibody testing," a statement read. "All necessary evaluations are under way in preparation for administering gene therapy. Throughout this process, Yaqeen's condition will be closely monitored and managed to maintain the highest standards of paediatric care." SMA is a condition that affects the motor neurons (nerve cells that transmit impulses to the muscles) and weakens the limbs. It makes walking difficult or impossible and creates problems with swallowing, as well as breathing. The treatment offered by Al Jalila Children's Hospital, called Zolgensma, is given once and is a form of gene therapy. It was approved by the US Food and Drug Administration four years ago and has been described as the most expensive drug in the world.
CBS News
28-05-2025
- Business
- CBS News
University of Michigan student on Medicaid worries about future amid looming federal cuts
President Trump's "big, beautiful bill," which passed in the House and is now awaiting a vote in the Senate, aims to reduce Medicaid federal spending by nearly $700 billion. Under the bill, millions of Americans could lose their Medicaid coverage. In Ann Arbor, University of Michigan student Vincent Pinti is earning a combined public policy and law degree. He uses a wheelchair due to a genetic condition called spinal muscular atrophy. When he graduated from high school, the West Virginia native searched for the most accessible campus in the country. "The University of Michigan was rated as the most accessible university in the United States," said Pinti. "And I had the notion to believe, well, if it's rated this highly, then all the supports and services that I would need would be in place here. Unfortunately, it wasn't the case." Pinti had to advocate for his needs and spent months navigating red tape to receive care under Medicaid, which differs from state to state. "I have 24/7 caregiving," he explained. "I have around 17 students that provide my care each week. Throughout my day I'll need anything from help getting out of bed in the morning, to going to the restroom, to having access to food, to having access to food. During the school year, they help make sure I can go to and from classes." Such round-the-clock care costs roughly $150,000 a year, but thanks to Medicaid, Pinti's needs are covered—for now. "The budget bill that was passed in the House this week will quite bluntly be catastrophic for people currently on the Medicaid program," said Pamela Herd, one of Pinti's professors. "You're looking at pretty massive cuts, a lot of additional complexities that are going to be added to an already complicated program. An expert in disability policy, Pinti says she understands the challenges he is facing now and in the future. "And the simple fact of the matter is they wanted to cut hundreds of billions of dollars from the program, and they've designed a bill that will do just that. And it's people like Vincent who are going to pay a high price for those cuts," she said. "With Medicaid, I might not be eligible for it going forward because it's really hard to simultaneously work and be on Medicaid," said Pinti. "When I finish college and I get a degree, Medicaid, it requires that you make under $30,000 a year. It says that you can't have more than $2,000 in assets at any given time. "So basically, I have a choice I have to make where I'll have to choose between having a full-time job and burning all my income on healthcare services or remaining on Medicaid and not having a career." Recent cuts to the U.S. Department of Education meant Pinti lost $20,000 in annual grants for students with disabilities. "We're trying to navigate a world where it seems like disability services and supports for our community are getting slashed left and right, and it doesn't seem like much thought is going into it," he said. "And real people, such as myself, are getting hurt."
