All articles tagged with #duchenne muscular dystrophy
Originally Published 26 days ago — by statnews.com
Doug Ingram of Sarepta Therapeutics is criticized as the worst biopharma CEO of 2025 for making risky decisions to push for broad approval of the gene therapy Elevidys for Duchenne muscular dystrophy, despite limited safety and efficacy data, leading to tragic consequences.
Originally Published 26 days ago — by U.S. Department of Health and Human Services (HHS) (.gov)
U.S. Secretary of Health and Human Services has approved adding Duchenne Muscular Dystrophy and Metachromatic Leukodystrophy to the newborn screening panel, enabling earlier diagnosis and treatment to improve outcomes for affected children.
Originally Published 2 months ago — by Investor's Business Daily
Sarepta Therapeutics' stock plummeted by over a third after reporting mixed Q3 results, including two drug failures and delayed test outcomes, despite strong sales of its gene therapy Elevidys. The company plans to seek traditional FDA approval for Amondys 45 and Vyondys 53, but uncertainties around regulatory and reimbursement issues persist. Meanwhile, sales of Elevidys exceeded expectations, though the outlook remains uncertain amid regulatory and demand challenges.
Originally Published 4 months ago — by The Independent
Two brothers with Duchenne muscular dystrophy received gene therapy, which significantly improved one brother's condition, but insurance denied coverage for the other, raising concerns about fairness and access to life-changing treatments.
Originally Published 4 months ago — by The Independent
Two brothers with Duchenne muscular dystrophy received gene therapy, which significantly improved one brother's condition, but insurance denied coverage for the other, citing lack of medical necessity, raising ethical and legal concerns for their family.
Originally Published 5 months ago — by The Economist
The FDA is currently facing turmoil after approving Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy in 2023, which was later linked to three patient deaths from liver failure, leading to a suspension request and a sharp decline in Sarepta's stock value.
Originally Published 5 months ago — by Yahoo Finance
Sarepta Therapeutics' shares rebounded after the FDA approved the resumption of shipments for its gene therapy Elevidys in the US for ambulatory patients with Duchenne muscular dystrophy, easing concerns about liquidity and market withdrawal, though shipments for non-ambulatory patients remain halted following recent deaths and regulatory scrutiny.
Originally Published 5 months ago — by statnews.com
The FDA has allowed Sarepta Therapeutics to resume shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, for younger patients who can walk, after a brief halt due to safety concerns related to non-ambulatory patients following two deaths. The agency continues to review the therapy for non-ambulatory patients, where a voluntary hold remains in effect.
Originally Published 5 months ago — by fda.gov
The FDA recommends lifting the voluntary hold on Elevidys for ambulatory patients with Duchenne Muscular Dystrophy after concluding that a recent death was unrelated to the therapy, while ongoing concerns for non-ambulatory patients remain under review.
Originally Published 5 months ago — by Sarepta Therapeutics
Sarepta Therapeutics issued a statement clarifying that the recent death of an eight-year-old Duchenne muscular dystrophy patient in Brazil, who received their gene therapy ELEVIDYS, was deemed unrelated to the treatment. The FDA is investigating the case, but Sarepta emphasizes their commitment to patient safety and regulatory transparency, highlighting the safety profile and risks associated with ELEVIDYS, including infusion reactions and liver injury.
Originally Published 5 months ago — by statnews.com
European regulators have decided not to approve the Duchenne muscular dystrophy gene therapy Elevidys, adding to global safety concerns and halting its distribution in Japan and Brazil, with ongoing investigations into safety issues.
Originally Published 5 months ago — by statnews.com
The halt of Sarepta Therapeutics' gene therapy Elevidys for Duchenne muscular dystrophy due to FDA safety concerns has caused heartbreak and uncertainty for families awaiting treatment, highlighting the emotional toll of regulatory decisions on young patients and their families.
Originally Published 5 months ago — by statnews.com
Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy, Elevidys, faces significant hurdles to return to the market after safety concerns, including liver injuries linked to patient deaths, making its re-approval challenging and potentially risking license revocation, according to a senior FDA official.
Originally Published 5 months ago — by CNBC
Sarepta Therapeutics announced it will temporarily pause all shipments of its Elevidys gene therapy in the U.S. following the death of a patient who received a different experimental treatment, after initially refusing to halt shipments despite regulatory requests.
Originally Published 5 months ago — by statnews.com
Sarepta Therapeutics has unexpectedly decided to pause all shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, following a request from the FDA, reversing its initial rejection of the agency's directive.