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Published on May 15, 2023
On Friday, an FDA advisory committee voted 8-6 that the benefits of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy (DMD) outweighs the treatment’s risks. If it is approved, SRP-9001 (delandistrogene moxeparvovec), which is being developed with Roche, will be the first gene therapy approved for this disease. The vote…
Published on February 27, 2023
A research team led by the University of Portsmouth in the U.K. has discovered that the Duchenne muscular dystrophy gene DMD is expressed differently in a range of different cancers in addition to causing the inherited degenerative muscle disease. As reported in the journal Cancers, they found that many cancerous…
Published on January 24, 2023
REGENXBIO is launching a Phase I/II trial of its gene therapy, RGX-202, for Duchenne muscular dystrophy. RGX-202 delivers a transgene for a novel microdystrophin protein that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 is designed for delivery and targeted expression of genes…
Published on March 11, 2022
A new trial may have pinpointed the first treatment that provides meaningful functional improvements in the most advanced cases of Duchenne muscular dystrophy (DMD). Researchers at UC Davis Health and six other sites showed that a cellular therapy appears to be safe and effective in stopping the deterioration of upper…
Published on December 22, 2021
The Phase Ib trial assessing Pfizer’s mini-dystrophin gene therapy candidate PF-06939926 in Duchenne muscular dystrophy (DMD) was placed on hold by the FDA after the company acknowledged the death of a young male participant in the open-label study. The study was designed to assess a single intravenous infusion of PF-06939926…
Published on December 1, 2021
Sponsored content brought to you by Debra Miller first noticed that something was wrong when her four-year-old son Hawken had trouble keeping up with the other kids while playing soccer. After two long years of hospital visits and inconclusive tests, they finally received a proper diagnosis of Duchenne muscular dystrophy,…
Published on October 7, 2021
Sarepta Therapeutics has announced the initiation of a new global study of SRP-9001 for the treatment of Duchenne Muscular Dystrophy (DMD). The study, known as EMBARK, will be carried out in partnership with Roche. SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue…
Published on August 27, 2021
Sponsored content brought to you by: Debra Miller first noticed that something was wrong when her four-year-old son Hawken had trouble keeping up with the other kids while playing soccer. After two long years of hospital visits and inconclusive tests, they finally received a proper diagnosis of Duchenne muscular dystrophy,…
Published on April 9, 2021
The genetic disorder Duchenne muscular dystrophy (DMD) is characterized by progressive muscle degeneration and weakness resulting from alterations of the protein dystrophin that helps keep muscle cells intact. Until recently, boys with DMD usually did not survive much beyond their teen years, but advances in cardiac and respiratory care have…
Published on November 5, 2020
A gene mutation in the cystic fibrosis gene may explain why some people with Duchenne Muscular Dystrophy (DMD) experience cardiac problems at an earlier stage to others according to research from the University of Texas Southwestern Medical Center, which could help clinicians to better personalize treatment for these patients. DMD…
Published on September 3, 2018
CRISPR-Cas9 has, for the first time, been tested by systemic delivery in a large animal—and the results are striking. Working in a dog model of Duchenne muscular dystrophy (DMD), the gene editing not only restored the expression of the protein dystrophin, it also improved muscle histology in the dogs. Eric…
Published on October 4, 2017
Scientists have developed a nonviral approach to delivering the CRISPR/Cas9 gene-editing system to cells that when tested in the mdx mouse model of Duchenne muscular dystrophy (DMD) repaired the faulty DMD gene, leading to improved strength and agility and reduced fibrosis. The platform, called CRISPR-Gold, uses gold nanoparticles to encapsulate…
Published on January 13, 2025
After spending over seven years at Sarepta Therapeutics working towards curing Duchenne muscular dystrophy (DMD) and other rare diseases, Bo Cumbo took the helm at a startup called AavantiBio to try to do something never done before—to target both the brain and the heart simultaneously with gene therapy. Now, this…
Published on November 20, 2024
REGENXBIO has already dosed the first patient in the next phase of the pivotal trial of its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD). Based on positive Phase I/II results, this week the company received FDA approval for expanding to a Phase I/II/III. They also received access to accelerated…
Published on November 15, 2024
Gene therapy for conditions such as muscular dystrophy has remained elusive because the large size of the gene prevents them from being packaged on the delivery vehicle used for most successful gene therapies—adeno-associated viruses (AAVs). But now, a team of researchers at the University of Rochester say they have developed…