Latest news with #Elevidys
Irish Independent
9 hours ago
- Health
- Irish Independent
Kilkenny granny running out of time to get lifesaving treatment for grandson (4) – ‘I will keep fighting until my or his last breath'
Diagnosed with Duchenne Muscular Dystrophy (DMD), William Moore's muscles have begun to waste away, not strong enough to hold up his small body. William will likely be fully wheelchair-bound by the age of eight, only four years from now. He has already started using a wheelchair part-time, proving just how rapidly the DMD is progressing and taking over his body. It is also unlikely that William will live long enough to celebrate a birthday beyond his 25th year – and that's best case scenario. DMD is a genetic disorder that causes progressive muscle weakness and deterioration that rapidly gets worse over time. As DMD progresses, the heart and respiratory muscles are affected as well. As a result, DMD is ultimately fatal. Most people diagnosed with the condition die at a young age from lung and heart issues caused by it. As there is no cure available for DMD, treatments focus instead on managing symptoms and improving the patient's quality of life. For William's grandmother, Sarka Palouckova, this feels like a jail sentence. She must sit back and watch helplessly as William continues to deteriorate right in front of her very eyes. Something as small as a slight gust of wind and sometimes even just thin air is enough to knock William over. Speaking to The Irish Independent in February of this year, Sarka explained that there is no treatment available in Ireland for William. "Back when William was first diagnosed with DMD and I realised there was no treatment for him in Ireland that could help him, I just kept crying and crying and crying,' said Sarka. 'I couldn't even work. I'm his grandmother, I want to protect him. ADVERTISEMENT Learn more "To this day, I still cry because I see how quickly the DMD is progressing. It's progressing at a faster pace than was expected and in comparison to other children the same age as him with DMD. "He is already starting to walk on his toes more and walking is getting harder for him and his balance is a lot worse. He falls over now because of just thin air.' After many sleepless nights scouring the internet, William's family came across a procedure called Elevidys, a gene therapy for people diagnosed with DMD. Elevidys prevents muscle cells from degenerating and may strengthen muscles by helping the body create protein. The only problem is the procedure is not available in Ireland or any EU country for that matter. Instead, William will have to travel all the way to America to the Boston Children's hospital if he wants to get Elevidys. However, the treatment will cost William's family an astronomical $3.7m, plus additional fees after he gets the treatment. A Go Fund Me page set up by William's grandmother has so far raised just over €39,000 out of the €4m target. While William's family are so grateful for everyone who has donated so far, William's condition is getting progressively worse, at a pace faster than anyone anticipated. Following a bad fall in May that left William unable to walk for over three weeks, Sarka is terrified her grandson is running out of time. "Recently, he had a fall in May and for three and a half weeks, he stopped walking altogether,' Sarka told The Irish Independent this week. 'It was a terrifying time, and I feared he might never walk again. "Thankfully, he's back on his feet now. In the meantime, he received his first wheelchair and a stand at home, which is helping him stretch his muscles.' As a result of the fall and his muscles wasting away, William can now only walk 300 metres before he falls over or has to be carried. When his legs are too exhausted to carry him further, William uses a wheelchair to get around. "The wheelchair is bittersweet,' said Sarka as she started to cry. 'In the wheelchair he has freedom to go wherever he likes without me carrying him which makes me so happy to see him happy and free. "But the wheelchair also makes me sad because it's a reminder of how bad William's condition is getting. We didn't expect him to be using a wheelchair already at just four-and-a-half years old.' Sarka does not know where to turn to next for help. With donations slowing down on the Go Fund Me page, she is at a loss of what to do next. "I cry most days. I feel so helpless and alone. I'm trying to save my grandson, but I don't know what else I can do. But I know I can't give up. I will keep fighting until mine or his last breath. "Some people might think there are many treatments available for Duchenne right now. But unfortunately, almost all of them are targeted at mutations from exon one to exon 58. William has a rare duplication of exon 62, which makes him ineligible for nearly every one of those options. "I've even contacted several clinical trials directly, but they all replied saying he's not eligible because of that specific mutation. Right now, the only available option for him is Elevidys in the US. That's why we're doing everything we can to try and get him there.' Despite the many setbacks, Sarak will keep fighting for William's future. With the help of all those who have donated so far to the Go Fund Me page and also other supporters of William who have organised their own fundraisers for the young Kilkenny boy, Sarka hopes she will eventually be able to raise the money for William's lifesaving treatment. At the moment, two young men from Kilkenny, Seán Lawlor and Cillian Larkin, are undertaking a mammoth cycle of approximately 4,000km from Dublin all the way to Istanbul to raise money for William's treatment. Seán and Cillian are currently cycling through Bosnia and Herzegovina. 'Seán and Cillian are two incredible guys from Kilkenny cycling from Dublin to Istanbul for William,' explained Sarka. 'They started their journey on May 16, but unfortunately about two weeks ago, while they were in Milan, Italy, they faced some difficulties. Cillian had his phone pickpocketed, and Sean's bicycle was stolen. "Thankfully, they managed to sort everything out after two days and are now back on track. "I also want to mention the support from local TD Natasha Newsome-Drennan who brought William's case to the Dáil twice,' added Sarka. 'I've sent many emails to all the TDs to stand with Natasha. Initially, it seemed positive and hopeful, but unfortunately, they have now gone completely silent. It's disheartening after such an encouraging start.' Also on Saturday July 5, Ryan's Bar in Kilkenny is hosting Cuckoo Fest 25 with live music from 3pm until late to raise money for William's treatment. "We are still fighting every single day to raise the money William needs,' said Sarka defiantly. 'At the same time, we're constantly reading, reaching out, and looking into any newly opened clinical trials that might offer hope. We will never give up.' You can donate to William Moore's Go Fund Me page to help raise money for his treatment here
Yahoo
25-06-2025
- Health
- Yahoo
Sarepta Therapeutics Is Already Down 84% This Year. And The Other Shoe Just Dropped.
Sarepta stock dropped again Wednesday after the FDA announced it's investigating the deaths of two patients who received Elevidys.
Yahoo
25-06-2025
- Business
- Yahoo
TD Cowen Downgrades Sarepta Therapeutics (SRPT) Stock to Hold, Reduces PT
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is one of the 10 Worst Aggressive Growth Stocks to Buy According to Short Sellers. On June 18, TD Cowen downgraded Sarepta Therapeutics, Inc. (NASDAQ:SRPT)'s stock to 'Hold' from 'Buy,' reducing the price objective to $24 from the prior target of $62, as reported by The Fly. This downgrade reflects the increased probability of withdrawal of Elevidys' FDA approval, for non-ambulatory and potentially ambulatory patients, as per the firm's analysis, mainly if the company continues to lose support from the Duchenne muscular dystrophy community. A laboratory technician in a white coat holding a microscope and examining a vial of biopharmaceuticals. The safety worries seem to be central to this downgrade, with the firm highlighting that Elevidys shows a 30% liver function test elevation rate. This suggests that the treatment's safety profile is being re-evaluated throughout all DMD patients. Overall, the new price objective for Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is considering the firm's DCF valuation of Elevidys, which is adjusted for the probability of market withdrawal. However, as part of a comprehensive review of safety data, Sarepta Therapeutics, Inc. (NASDAQ:SRPT) continues to take proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. Overall, the company stated that the broader biotech market witnessed significant pressure in Q1 2025. Considering the company's significant revenue and a deep pipeline, it is well-placed to navigate the challenging times. Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company focusing on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for treating rare diseases. While we acknowledge the potential of SRPT to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than SRPT and that has 100x upside potential, check out our report about this cheapest AI stock. READ NEXT: 13 Cheap AI Stocks to Buy According to Analysts and 11 Unstoppable Growth Stocks to Invest in Now Disclosure: None. Sign in to access your portfolio
The Sun
25-06-2025
- Health
- The Sun
FDA probes deaths linked to Sarepta's gene therapy for muscular dystrophy
THE U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver failure in non-ambulatory Duchenne muscular dystrophy patients after receiving Sarepta Therapeutics' gene therapy, Elevidys. In June, Sarepta reported a second death in a patient who had received its gene therapy, which raised concerns about the safety and future demand for the treatment. The patients who died were a 16-year-old, weighing 70 kilograms, and a 15-year-old, weighing 50 kilograms. Both boys were non-ambulatory and their deaths occurred within 90 days after treatment, the company said in an investor call last week. The two patients showed signs of acute liver failure and were hospitalized less than two months after treatment with Elevidys, the health regulator said on Tuesday. It added that it is evaluating the need for further regulatory action. Sarepta said in an email to Reuters, 'The FDA communication was triggered by our report to the FDA and our suggested update to the label to include information relating to the recent events.' The U.S. prescribing information for Elevidys warns of acute liver injury but does not mention liver failure or death. Elevidys, approved by the FDA in 2024 for ambulatory Duchenne muscular dystrophy patients aged four and older, is the only gene therapy available for the disease. It carries a known risk of liver damage and was conditionally approved for non-ambulatory patients despite failing to meet the main goal in a late-stage study. Sarepta said earlier this month it was investigating both cases independently and in relation to each other to identify any common risk factors. The company has suspended its Elevidys sales forecast for 2025, halted shipments to non-ambulatory patients and is working with regulators on a new treatment plan.
Yahoo
24-06-2025
- Health
- Yahoo
FDA investigates patient deaths after treatment with Sarepta's gene therapy
(Reuters) -The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver failure in non-ambulatory Duchenne muscular dystrophy patients after receiving Sarepta Therapeutics' gene therapy, Elevidys. In June, Sarepta reported a second death in a patient who had received its gene therapy, which raised concerns about the safety and future demand for the treatment. The patients who died were a 16-year-old, weighing 70 kilograms, and a 15-year-old, weighing 50 kilograms. Both boys were non-ambulatory and their deaths occurred within 90 days after treatment, the company said in an investor call last week. The two patients showed signs of acute liver failure and were hospitalized less than two months after treatment with Elevidys, the health regulator said on Tuesday. It added that it is evaluating the need for further regulatory action. Sarepta said in an email to Reuters, "The FDA communication was triggered by our report to the FDA and our suggested update to the label to include information relating to the recent events." The U.S. prescribing information for Elevidys warns of acute liver injury but does not mention liver failure or death. Elevidys, approved by the FDA in 2024 for ambulatory Duchenne muscular dystrophy patients aged four and older, is the only gene therapy available for the disease. It carries a known risk of liver damage and was conditionally approved for non-ambulatory patients despite failing to meet the main goal in a late-stage study. Sarepta said earlier this month it was investigating both cases independently and in relation to each other to identify any common risk factors. The company has suspended its Elevidys sales forecast for 2025, halted shipments to non-ambulatory patients and is working with regulators on a new treatment plan.
