Asklepios BioPharmaceutical (AskBio) is planning to launch a Phase 1/2 clinical study to evaluate LION-101, its investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Dosing in the trial is expected to start in the first half of next year. “We are excited to begin clinical trials…
News
For the first time, a new mouse model has been shown to mimic the heart disease features — specifically, to progress into late-stage heart failure, similar to humans — of people with Duchenne muscular dystrophy (DMD), a study reports. Moreover, using the new mouse model, researchers gathered promising data…
Sarepta Therapeutics‘ investigational gene therapy SRP-9001 showed early signs of biologic efficacy in muscle biopsies from 11 boys with Duchenne muscular dystrophy (DMD) treated in a Phase 1 clinical trial, the company announced in a press release. “Robust” expression of micro-dystrophin was evident in cells at 12…
Two more participants have been dosed in the IGNITE DMD clinical trial, bringing to eight the total number of Duchenne muscular dystrophy (DMD) patients thus far given Solid Biosciences‘ investigational gene therapy SGT-001. The U.S. trial is testing SGT-001’s safety and efficacy in boys with DMD ages…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to losmapimod, a potential treatment for facioscapulohumeral muscular dystrophy (FSHD) being developed by Fulcrum Therapeutics. The designation is intended to help bring effective treatments for serious conditions to market more quickly. It grants Fulcrum, as…
Jesse’s Journey, a Canadian nonprofit founded by parents of a child with Duchenne muscular dystrophy (DMD), has given a total CA$1.7 million to support four projects looking to protect muscle fibers, or treat patients through gene editing and stem cells approaches. The award, worth about $1.4 million, is…
Pfizer has announced a delay in opening U.S. sites for its Phase 3 clinical trial CIFFREO, evaluating the investigational gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The delay is caused by questions…
Sarepta Therapeutics has announced positive results from a global study evaluating its investigational treatment SRP-5051 in people with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Results from part A of the…
Ray Jordan and Cyndi Segroves, a married couple with facioscapulohumeral muscular dystrophy (FSHD), haven’t let their disability get in the way of anything. They’ve traveled from their Arizona home to destinations around the world — from Hawaii and New…
The muscles of people with Duchenne muscular dystrophy (DMD) show profound impairment, with changes in fatty molecules and energy metabolism, as compared with those of their healthy peers, a small French study revealed. While previous studies have reported such metabolic changes in DMD patients, this was the first…
Recent Posts
- MDA-led project to map how human muscles repair themselves
- My willingness to educate others about MD benefits all of us
- The unbearable weight of grief that smoothes the jagged edges
- New analysis tracks Elevidys outcomes 3 years after treatment
- Still playing my tune with a little help from my friends