News Medical Duchenne Muscular Dystrophy News Feed Latest Duchenne Muscular Dystrophy News and Research
- New method yields up to twice as many therapeutic myogenic cells as previous protocols
on October 30, 2025 at 6:59 pmIf cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body’s best interests, then degenerative diseases are disorders of deprivation.
- Under Trump, FDA seeks to abandon expert reviews of new drugs
on September 13, 2025 at 2:41 amFDA leaders under President Donald Trump are moving to abandon a decades-old policy of asking outside experts to review drug applications, a move critics say would shield the agency’s decisions from public scrutiny.
- Improving gene therapy safety with human kidney organoids
on August 19, 2025 at 11:57 amRyuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, “AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids.”
- Researchers solve longstanding mystery of protein release in cells
on August 6, 2025 at 3:56 amIt’s known as biology’s central dogma: All living organisms’ genetic information is stored in DNA, which is transcribed into RNA, which is translated into proteins that perform nearly all essential tasks in a cell.
- Marbling of fat inside muscles found to block healing and weaken strength
on July 28, 2025 at 5:23 pmConsider the delicate web of fat in a Wagyu steak. The “marbling” that makes carnivore connoisseurs swoon is a visual heuristic for quality flavor.
- Targeting GLUD1 shows promise in restoring muscle function in Duchenne muscular dystrophy
on July 17, 2025 at 5:29 amNew research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD).
- New cellular entry pathway improves gene therapy outcomes
on July 15, 2025 at 8:04 amScientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy, Pompe disease and hemophilia.
- Improving RNA drug delivery through intracellular traffic control
on July 1, 2025 at 8:13 amA recent study involving researchers from the University of Basel reveals that slowing down the intracellular transport of RNA-based drugs can significantly enhance their effectiveness.
- AAN releases Evidence in Focus Article on new gene therapy for Duchenne muscular dystrophy
on May 15, 2025 at 3:25 amTo help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, published May 14, 2025, online in Neurology.
- Existing therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy
on May 1, 2025 at 4:01 amA new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a severe condition that leads to muscle weakness and premature death.
HOSPITAL AND RESEARCHERS AWARDED $2.2M FOR DUCHENNE GENE THERAPY INVESTIGATION
September 9, 2017
The nonprofit Duchenne muscular dystrophy (DMD) research and advocacy organization Parent Project Muscular Dystrophy (PPMD) awarded a $2.2 million grant to Jerry Mendell, MD, PhD; co-principal investigator Louise Rodino-Klapac, PhD; and Nationwide Children’s Hospital in Columbus, Ohio, where they both work.
The funding will support Mendell, Rodino-Klapac, and their research team’s investigation into gene therapy as a potential Duchenne MD treatment.
Mendell is a professor of pediatrics and neurology and director of the center for gene therapy at Nationwide Children’s Hospital’s Research Institute, and Rodino-Klapac heads the Rodino-Klapac Gene Therapy Lab for Muscular Dystrophies, which is focused on developing gene therapy-based vectors for the treatment of various neuromuscular disorders. She is also an assistant professor at the The Ohio State University School of Medicine.
November 11, 2016
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.
October 6, 2016
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory nonsense mutation Duchenne muscular dystrophy patients (nmDMD). The results, which are being presented today as part of a company-sponsored symposium, are based on PTC’s analyses of lung function data from one of PTC’s ongoing open-label extension studies (Study 019) versus natural history data from a comparable non-ambulatory cohort.
September 28, 2016
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the first patient dosed in the phase III clinical trial of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.
September 19, 2016
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for EXONDYS 51™ (eteplirsen) as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping.
December 18, 2015
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen, on January 22, 2016. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the eteplirsen NDA is February 26, 2016.
December 18, 2015
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) has notified the Company that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (PDUFA) action date for KyndrisaTM (drisapersen) of December 27, 2015, and anticipate taking action in early January 2016.
SPELL CHECKING NATURE: VERSATILITY OF CRISPR/CAS9 FOR DEVELOPING TREATMENTS FOR INHERITED DISORDERS
December 10, 2015
Clustered regularly interspaced short palindromic repeat (CRISPR) has arisen as a frontrunner for efficient genome engineering. How- ever, the potentially broad therapeutic implications are largely unexplored.
November 24, 2015
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.