Latest news with #DuchenneMuscularDystrophy
Yahoo
2 hours ago
- Business
- Yahoo
Capricor Therapeutics announces four-year data from HOPE-2 OLE study
Capricor Therapeutics (CAPR) announced four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension study of Deramiocel, the Company's lead cell therapy candidate for Duchenne Muscular Dystrophy. The data will be featured in the session titled 'Therapies that Slow Progression' at the Parent Project Muscular Dystrophy 2025 Annual Conference, taking place June 21, 2025, in Las Vegas, Nevada. After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline. Further, a subgroup analysis of patients with baseline LVEF greater than45% showed an even greater clinical benefit, supporting early intervention with Deramiocel to potentially preserve cardiac function. Additionally, treatment continued to slow skeletal muscle disease progression, as measured by Performance of the Upper Limb, with patients experiencing a smaller average decline in the fourth year compared to the first year. Together, these findings suggest that extended treatment with Deramiocel may help attenuate the progression of DMD over time. Deramiocel continues to maintain a favorable safety profile throughout the study. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on CAPR: Disclaimer & DisclosureReport an Issue FDA's Nicole Verdun placed on administrative leave, STAT reports Capricor Therapeutics announces ODD to Deramiocel Capricor treatment of Becker muscular dystrophy granted orphan designation Roth thinks Capricor Therapeutics drug could still be approved by August 31 Adobe reports Q2 beat, Meta invests in Scale AI: Morning Buzz Sign in to access your portfolio
CBS News
13 hours ago
- 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."
Daily Mirror
4 days ago
- Health
- Daily Mirror
'My son, 12, can't have free life-changing drug because of where we live'
No mother should have to watch their child suffer, but one woman shares what it's like witnessing her 12-year-old boy slowly deteriorate because he's not eligible for a new drug that could improve his life Having to watch her son slowly fade away is heartbreaking in itself but knowing he can't have a free drug because of where he lives, is nothing less than mental torture. That's the everyday reality for Colleen who is mum to Alfie Pentony, 12, from Newry, County Down, who was diagnosed with Duchenne Muscular Dystrophy (DMD) when he was just four years old. Her fun-loving, football -mad son is getting weaker every day - but a new drug, Givinostat, could slow down his incurable condition and help him walk for another three years. But although the drug is free in other parts of the UK, the Belfast Trust has said it does 'not have the capacity' to offer it in Northern Ireland. 'We're heartbroken and devastated,' Colleen, 43, tells The Mirror. 'It's unjust, shameful and feels like mental torture knowing there's a drug that could help him but he can't have it because of where we live. "Alfie is a typical little boy who wants to grow up and play for Newcastle United like Lewis Miley. We know this is never going to happen but if he got this drug, it would at least help prolong his ability to walk for maybe another three years and continue to kick a ball for longer.' Muscular Dystrophy is an inherited genetic condition that gradually causes the muscles to weaken so sufferers eventually lose the ability to walk - with most only living to their 20s or 30s. While DMD is one of the most common and severe forms - this new drug could slow it down giving patients an extra three years without being restricted to a wheelchair. 'We don't know how long Alfie has left on his feet, most boys are wheelchair bound at the age of 12, so he is beating the odds so far,' Colleen adds. 'His muscles are breaking down every day - but time is muscle and in a few months it could be too late for him. "Every day that he doesn't have access to the drug, he is getting weaker than the boys who have access to the drug which is unjust and cruel. The Trust has put a price tag on my son's life and I'm not having it, I don't know how they can sleep at night. 'This drug is something that could dramatically change the course of our son's disease and to be told we can't have it is mental torture. I feel let down by the service that is meant to protect these boys, the fact that we are the only part of the UK that won't provide this drug is shameful. It could massively prolong Alfie's life and his quality of life.' Alfie is also pleading for the drug - not just for himself - but for the 10 other boys in Northern Ireland who it could also help. The anti-inflammatory drug, Givinostat, has been approved for use in the UK by the medicines regulator and is available on the NHS for people who meet strict criteria. But individual trusts must apply to take part in the Early Access Programme (EAP). The Belfast Trust said currently it 'is not in a position to proceed' to offer the free drug. 'Its implementation will need to be managed within defined and agreed protocols and additional staffing resources will also be required to ensure the treatment can be provided safely," a spokesperson added. "We recognise this will be very disappointing for families and the Belfast Trust sincerely apologises to them.'
Forbes
4 days ago
- Business
- Forbes
SRPT Stock: What's Happening With Sarepta Therapeutics?
CANADA - 2025/06/08: In this photo illustration, the Sarepta Therapeutics logo is seen displayed on ... More a smartphone screen. (Photo Illustration by Thomas Fuller/SOPA Images/LightRocket via Getty Images) Sarepta Therapeutics (NASDAQ: SRPT) suffered a devastating blow on Monday, June 16 when its stock plummeted 42% following news of a second patient death linked to its Duchenne muscular dystrophy gene therapy, Elevidys. The biotechnology company and its global partner Roche immediately suspended commercial and clinical use of the treatment in non-ambulatory patients pending a comprehensive safety review. The decision came after Roche conducted a safety profile reassessment that revealed concerning patterns following the two fatalities. Commercial distribution has ceased effective immediately, while enrollment in ongoing clinical trials has been paused until additional risk mitigation measures can be implemented in study protocols. Now, if you are looking for an upside with a smoother ride than an individual stock, consider the High-Quality portfolio, which has outperformed the S&P, and clocked >91% returns since inception. On a separate note, see – Should You Buy CRWV Stock After A Whopping 4x Rise? Elevidys represents a significant advancement in gene therapy for Duchenne Muscular Dystrophy, a devastating genetic disorder that progressively weakens muscles. The FDA granted accelerated approval in June 2023 for ambulatory patients aged 4-5, later expanding traditional approval to all ambulatory patients aged 4 and older. However, continued approval for non-ambulatory patients remained contingent on confirmatory clinical trial results. The financial implications for Sarepta are profound. Elevidys generated 43% of the company's total revenues last year, making it the cornerstone of Sarepta's commercial portfolio. Industry analysts had projected peak sales of over $3 billion for the therapy, expectations that now face substantial revision given the restricted patient population. Sarepta's financial profile already presented significant challenges before this setback. Despite achieving average revenue growth exceeding 40% over the past three years, the company remains unprofitable with an operating cash flow margin of negative 25% and a net income margin of negative 11%. The company carries $1.3 billion in debt against a current market capitalization of $2.06 billion, resulting in a concerning debt-to-equity ratio of 63%. The stock's trajectory illustrates the extreme volatility inherent in pharmaceutical investments. Shares have collapsed from their 52-week high of approximately $172 to current levels around $20, representing the kind of dramatic price swings that characterize companies heavily dependent on revenues from only a few drugs. While Sarepta's multi-year low valuations might appear attractive to contrarian investors, the investment proposition carries substantial risks. The combination of recent safety concerns with its flagship product, overwhelming dependence on a single revenue source, significant cash burn, and extreme stock volatility creates a highly speculative investment scenario suitable only for investors with high risk tolerance. Investors seeking exposure to innovative healthcare developments might consider diversified approaches, such as the Trefis High Quality (HQ) Portfolio, that spread risk across multiple companies and sectors, rather than concentrating capital in individual speculative positions. With a collection of 30 stocks, this portfolio has a track record of comfortably outperforming the S&P 500 over the last 4-year period. Why is that? As a group, HQ Portfolio stocks provided better returns with less risk versus the benchmark index; less of a roller-coaster ride, as evident in HQ Portfolio performance metrics.
Washington Post
5 days ago
- Business
- Washington Post
Biotech pauses trial after second patient death linked to gene therapy
Biotech firm Sarepta Therapeutics said that a second patient died after receiving its gene therapy to treat Duchenne muscular dystrophy, the latest drug to draw scrutiny after winning fast regulatory approval. The Cambridge, Massachusetts-based firm's stock lost more than 40 percent of its value Monday, a day after disclosing the death and announcing that it would temporarily stop shipping the treatments for certain patients, and pause a clinical trial, while it evaluates its protocols.
