Topic
Duchenne muscular dystrophy
8 stories related to this topic, newest first.
Statscience38 days ago
Regenxbio Gene Therapy for Duchenne Muscular Dystrophy Succeeds in Trial
Regenxbio announced on May 14, 2026 that its experimental treatment produced high levels of a miniaturized muscle protein, clearing the path for an FDA submission. The Rockville, Md.-based company aims to offer a safer and more effective option than Sarepta Therapeutics’ Elevidys…
prnewswire.comscience44 days ago
Capricor Therapeutics Sues Nippon Shinyaku and NS Pharma Over Pricing Dispute on Duchenne Muscular Dystrophy Therapy Deramiocel
Capricor Therapeutics filed suit in New Jersey state court on May 8, 2026, accusing Nippon Shinyaku and its U.S. subsidiary NS Pharma of failing to execute marketing plans for deramiocel and refusing to correct a pricing formula flaw discovered in March 2025.
The Independentworld44 days ago
NHS England to Roll Out Givinostat for Eligible Duchenne Muscular Dystrophy Patients
Givinostat will become available to around 530 eligible patients in England with Duchenne muscular dystrophy following a commercial agreement between its manufacturer and NHS England. The National Institute for Health and Care Excellence confirmed the decision after the company r…
prnewswire.comscience45 days ago
Entrada Duchenne Therapy Falls Short in Early Trial
Entrada Therapeutics reported that its next-generation exon-skipping drug for Duchenne muscular dystrophy did not meet expectations in an early-stage study. The company is among several developing improved treatments aimed at increasing functional dystrophin production in patient…
Substrate placeholder — needs reviewscience52 days ago
Roche to Run New Elevidys Trial for European Approval; FDA Plans Advisory Panels on Compounded Peptides
Roche announced a new trial of Elevidys, Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy, to pursue approval in Europe. Meanwhile, the FDA will hold advisory panel meetings starting July 2026 to reconsider restrictions on compounded peptides.
Substrate placeholder — needs reviewscience74 days ago
Biotech Developments Include Duchenne Drug Class, Gilead Acquisitions and Industry Updates
A class of drugs for Duchenne muscular dystrophy has drawn attention in recent discussions. Gilead Sciences continues its series of mergers and acquisitions in the biotech sector. Additional news covers ongoing advancements and regulatory matters in biotechnology.
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Substrate placeholder — needs reviewscience74 days ago
Elevidys Gene Therapy Shows Positive Results in Duchenne Muscular Dystrophy Trial
Sarepta Therapeutics reported positive topline results from a phase 3 trial of its gene therapy Elevidys for Duchenne muscular dystrophy. The trial met its primary endpoint of improved motor function in patients aged 4 to 7. The Duchenne muscular dystrophy field has experienced s…
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Substrate placeholder — needs reviewscience74 days ago
Recent Health News Highlights Breakthrough in Duchenne Treatment and Insulin Price Cap
A new therapy shows promise for Duchenne muscular dystrophy patients. Insulin prices are capped at $35 for many users. Additional evidence supports over-the-counter availability of abortion medications.
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