News Medical Duchenne Muscular Dystrophy News Feed Latest Duchenne Muscular Dystrophy News and Research
- New mRNA technique targets disease cells while sparing healthy oneson February 19, 2025 at 11:36 am
Imagine a breakthrough in cancer treatment where only malignant cells are targeted, sparing healthy host cells; or patients with abnormal protein synthesis are treated to produce a healthy protein.
- Novel drug holds promise for treating Duchenne muscular dystrophyon December 16, 2024 at 6:25 pm
A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.
- Harnessing exosomes to deliver therapeutic proteinson December 12, 2024 at 4:43 am
Researchers at The Ottawa Hospital and the University of Ottawa have discovered an 18-digit code that allows proteins to attach themselves to exosomes – tiny pinched-off pieces of cells that travel around the body and deliver biochemical signals.
- SRF awards $230,000 grant to advance research on SYNGAP1-Related Disorderson December 4, 2024 at 2:03 pm
The SynGAP Research Fund (SRF) has awarded a $230,000 grant to The Jackson Laboratory Rare Disease Translational Center to characterize a mouse model for SYNGAP1-Related Disorders, (SRD), a severe neurodevelopmental disorder marked by seizures, intellectual disabilities, and motor and behavioral challenges.
- StitchR technology delivers large genes for muscular dystrophy treatmenton November 15, 2024 at 2:54 am
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.
- CRISPR-Cas13 emerges as a game changer in RNA-targeted therapieson September 25, 2024 at 7:30 pm
In recent years, the scientific community has made significant strides in the field of gene editing, particularly through the development of the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) systems.
- New tool shows promise for assessing neurobehavioral difficulties in children with Duchenne muscular dystrophyon August 14, 2024 at 7:50 pm
A pilot study using a new screening tool shows promise for assessing neurobehavioral difficulties in children with Duchenne muscular dystrophy (DMD) and improving quality of life for patients and their families.
- Breakthrough gene therapy restores dystrophin protein in Duchenne muscular dystrophyon July 25, 2024 at 3:39 am
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).
- UH research identifies key mechanisms of skeletal muscle regenerationon July 23, 2024 at 12:06 am
Newly published research from the University of Houston College of Pharmacy identifies key mechanisms of skeletal muscle regeneration and growth of muscles following resistance exercise.
- Breakthrough gene therapy offers hope for Duchenne muscular dystrophyon July 18, 2024 at 12:21 pm
A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.
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.