Latest news with #muscularDystrophy
BBC News
03-07-2025
- Politics
- BBC News
Welfare needs reform despite U-turn
A grandmother living with a degenerative disease says the government's U-turn on personal independence payment (Pip) will still not help those in need, as "complete reform" is Thomas, 59, from Bulkington, near Bedworth, has had muscular dystrophy for nearly 30 years, receiving disability benefits ever since. On Tuesday, the Labour government said it would not change Pip rules until the recommendations of a review could be implemented, having won a vote on its benefits bill following last-minute concessions to party rebels. However, Ms Thomas said that despite some of the benefit cuts being reversed, the "one size fits all" welfare system remained "broken". She is no longer able to walk and is cared for primarily by her husband, but said the current system categorised her in the same group as a person who "had some difficulty walking around"."Others get the same level as me, but have half the disability. It's a constant fight to survive," she government needed to reclassify "the impacts of disabilities" in order for it to improve, Ms Thomas said. She said it was "upsetting" to have to ask for help, but any cut to Pip would be "devastating". The government previously announced current claimants would be protected from stricter eligibility changes to disability assessments have been proposed for anyone claiming Pip for the first time after the recommendations of a current review are implemented. The proposals require those applying to score at least four points for a single activity, rather than across a range of different ones, with assessments scoring everyday activities, such as washing hair, from zero to 12, for no difficulty to most difficulty. "There needs to be complete reform not just an alteration to Pip points," Ms Thomas said. 'Divide in the community' The government previously defended the proposals, after it pointed to steep rises in the numbers claiming benefits and said making changes was the only way to ensure the system remained sustainable in the Mel Smith, from the Coventry charity Grapevine that supports people with special needs, said the proposed reforms still raised concerns."What does that do for the disabled community, you'll get one group who will be eligible and then other people who won't," she argued the new criteria was therefore "unfair" and future claimants would "lose out on vital support". Follow BBC Coventry & Warwickshire on BBC Sounds, Facebook, X and Instagram.
CBS News
21-06-2025
- Health
- CBS News
North Texas family raising awareness of muscular dystrophy struggles
With his face focused on the screen, Brandon Hale's fingers moved like lightning as he created masterpieces in Minecraft. "I'm currently building a house," the typically shy 16-year-old said when gently asked. "Took me two years to get good." He is bright and funny, but Brandon's body is slowly betraying him. He was diagnosed with Duchenne muscular dystrophy at age 5. Family remembers the day everything changed "I can remember the day he got diagnosed," said his older sister, Emily. "I can remember sitting on the stairs while my mom was on the phone with the doctor, and then the air in the room — the air disappeared from the room the second we kind of found out." Duchenne is a severe form of muscular dystrophy caused by the body's inability to produce dystrophin, a protein essential for muscle health. Over time, all muscles — legs, arms, lungs, and heart — deteriorate. CBS News Texas Living in the moment, despite the odds "We just try to take it one day at a time and do the best we can and enjoy every day," said Brandon's mother, Margaret Hale. "Because I know when you think about the big picture — that it's a degenerative disease and it's going to cost him his life at some point." Still, Margaret refuses to lose hope. "But you never know. I mean, you don't have tomorrow necessarily," she said. "So, you've got to make the best of what you've got right now." Father's health adds to family's burden Even "right now" is hard. Brandon's father, Eric Hale, is in a Denton rehab hospital. A few weeks ago, he woke up with a swollen foot. It led to a below-the-knee amputation due to complications from diabetes. "I just want to bring awareness to it — the struggles, the everyday struggles," Eric said. And not just his own, but Brandon's and those of other families navigating this disease. Eric and Margaret both grew up watching the Jerry Lewis Labor Day MDA telethon. When it ended in 2014, he said, much of the awareness disappeared—but the disease did not. Financial strain and lack of support "You know, most people probably think we get assistance, and we don't," Eric said. "The only time we've ever gotten help was when he first started taking the steroids. If we had to pay for them every month until we hit our deductible, they'd be almost $5,000 a month." Eric said he speaks for "all the families falling through the cracks of a broken system" — earning too much to qualify for significant assistance, but not enough to meet their son's growing needs. "We've drained our savings. We've drained our retirement," he said. "Just to provide for my kids. And again, it's a struggle every single day. I want — both my kids — but to make his life as comfortable as possible for as long as he's got." A father's love and unspoken fears Eric called his teenage son "my hero." "Both my kids are. But the strength that that little boy has..." His voice quivered. "We haven't talked about it. But I'm smart enough to know that he's done his own research on it. I have no doubt. But we've never had that conversation with him because I can't." He paused, overcome with emotion. Home not built for disability The emotional toll must wait. Each day, the Hales face practical challenges, like a home that isn't accessible. "The doorways are just not wide enough for him to even get through in the wheelchair," Margaret said. "A roll-in shower for him. And then, just maybe widening some of the doorways to help make it easier to get him in and out. Those are the real major things we would want to have done if we could." Family sacrifices to stay together So, day to day, they do the best they can. Margaret, who built a career in education, is now a full-time caregiver and homeschool teacher. She's looking for a part-time, remote job to help support the family. Emily was accepted into Texas Tech but is postponing that dream to stay home and help. "I mean, that's my family," Emily said. "I would do anything for them. So if that means me working the next couple of years of my life, I mean, it's not the end of the world to try to figure out what's next while still being here for them." Focusing on today, not tomorrow Even if "what's next" is simply enduring the "right now." "I don't think about 10 years from now," Eric said. "I think about a month from now, two weeks from now, tomorrow. That's all I can do."
Al Arabiya
16-06-2025
- Health
- Al Arabiya
Second Patient Death Reported With Gene Therapy for Muscular Dystrophy
Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the death over the weekend and provided additional details about its response, which includes pausing shipments of the therapy Elevidys for older patients who are no longer able to walk. The one-time treatment is approved for children with a genetic variant of Duchenne's muscular dystrophy, which causes weakness, loss of mobility, and early death in males. Elevidys is the first gene therapy approved in the US for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. The second death, like an earlier one reported in March, occurred in a teenage boy who suffered a fatal case of acute liver injury, a known side effect of the therapy. Older patients receive a larger dose of the therapy. Sarepta said it would pause a study in those patients and assemble an expert panel to recommend new safety protocols for taking the drug. Those changes are expected to include increased use of immune-system-suppressing drugs, company executives said Monday. The liver injury associated with the therapy is thought to be connected to the immune system's response. Sarepta said it was cooperating with the Food and Drug Administration, which would have to sign off on any changes to the product's use. Elevidys received expedited approval despite concerns from some FDA scientists about its effectiveness in treating Duchenne's. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older regardless of whether they are still able to walk. Previously, it was only available for younger patients who were still walking. Shares of the Cambridge, Massachusetts–based company fell more than 42 percent to close at about 21 in trading. Wall Street analysts speculated that FDA officials, including new vaccine chief Dr. Vinay Prasad, might impose more restrictions on the drug or even block its use. Prasad has been highly critical of the therapy since its approval under the agency's previous leadership. 'Now with two deaths reported in this segment of the market, it seems incrementally more possible that the FDA could step in and remove the therapy from the market in non-ambulatory patients,' said Leerink Partners analyst Joseph Schwartz in a Sunday research note. Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin into patient cells. It costs $3.2 million for a one-time infusion.
The Independent
16-06-2025
- Health
- The Independent
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the death over the weekend and provided additional details about its response, which includes pausing shipments of the therapy, Elevidys, for older patients who are no longer able to walk. The one-time treatment is approved for children with a genetic variant of Duchenne's muscular dystrophy, which causes weakness, loss of mobility and early death in males. Elevidys is the first gene therapy approved in the U.S. for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. The second death, like an earlier one reported in March, occurred in a teenage boy who suffered a fatal case of acute liver injury, a known side effect of the therapy. Older patients receive a larger dose of the therapy. Sarepta said it would pause a study in those patients and assemble an expert panel to recommend new safety protocols for taking the drug. Those changes are expected to include increased use of immune-system suppressing drugs, company executives said Monday. The liver injury associated with the therapy is thought to be connected to the immune system's response. Sarepta said it was cooperating with the Food and Drug Administration, which would have to sign off on any changes to the product's use. Elevidys received expedited approval despite concerns from some FDA scientists about its effectiveness in treating Duchenne's. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, regardless of whether they are still able to walk. Previously it was only available for younger patients who were still walking. Shares of the Cambridge, Massachusetts-based company fell more than 42% to close at about $21 in trading. Wall Street analysts speculated that FDA officials, including new vaccine chief Dr. Vinay Prasad, might impose more restrictions on the drug or even block its use. Prasad has been highly critical of the therapy since its approval under the agency's previous leadership. 'Now with two deaths reported in this segment of the market, it seems incrementally more possible that the FDA could step in and remove the therapy from the market in non-ambulatory patients,' said Leerink Partners analyst Joseph Schwartz, in a Sunday research note. Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin into patient cells. It costs $3.2 million for a one-time infusion. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Science and Educational Media Group and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Associated Press
16-06-2025
- Health
- Associated Press
Second patient death reported with gene therapy for muscular dystrophy
WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the death over the weekend and provided additional details about its response, which includes pausing shipments of the therapy, Elevidys, for older patients who are no longer able to walk. The one-time treatment is approved for children with a genetic variant of Duchenne's muscular dystrophy, which causes weakness, loss of mobility and early death in males. Elevidys is the first gene therapy approved in the U.S. for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. The second death, like an earlier one reported in March, occurred in a teenage boy who suffered a fatal case of acute liver injury, a known side effect of the therapy. Older patients receive a larger dose of the therapy. Sarepta said it would pause a study in those patients and assemble an expert panel to recommend new safety protocols for taking the drug. Those changes are expected to include increased use of immune-system suppressing drugs, company executives said Monday. The liver injury associated with the therapy is thought to be connected to the immune system's response. Sarepta said it was cooperating with the Food and Drug Administration, which would have to sign off on any changes to the product's use. Elevidys received expedited approval despite concerns from some FDA scientists about its effectiveness in treating Duchenne's. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, regardless of whether they are still able to walk. Previously it was only available for younger patients who were still walking. Shares of the Cambridge, Massachusetts-based company fell more than 42% to close at about $21 in trading. Wall Street analysts speculated that FDA officials, including new vaccine chief Dr. Vinay Prasad, might impose more restrictions on the drug or even block its use. Prasad has been highly critical of the therapy since its approval under the agency's previous leadership. 'Now with two deaths reported in this segment of the market, it seems incrementally more possible that the FDA could step in and remove the therapy from the market in non-ambulatory patients,' said Leerink Partners analyst Joseph Schwartz, in a Sunday research note. Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin into patient cells. It costs $3.2 million for a one-time infusion. ___ The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Science and Educational Media Group and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
