Nanoparticles deliver microRNAs to muscle stem cells for potential muscular dystrophy treatment
In an international collaboration, researchers have made an important breakthrough in the therapeutic delivery of microRNAs ...
Pharmaceutical Technology5h
FDA removes clinical hold on Entrada’s Duchenne therapy
Entrada Therapeutics can plough on with the development of its Duchenne muscular dystrophy (DMD) therapy, ENTR-601-44, now ...
STAT7d
Duchenne gene therapy from Solid Biosciences produces promising results in early study
Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...
GlobalData on MSN9h
Success for YolTech’s hyperoxaluria in vivo gene therapy in early-stage trial
YolTech's gene therapy is intended as a one-time life-long cure for patients living with PH1, which can lead to kidney ...
Solid says early data suggest ‘differentiated’ Duchenne gene therapy
The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...
FierceBiotech1d
Entrada's 2-year ordeal ends as FDA lifts hold on DMD drug
Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA ...
Health.com5d
What To Know About Duchenne Muscular Dystrophy (DMD)
Physical therapy can help maintain muscle ... MedlinePlus. DMD gene. MedlinePlus. Duchenne muscular dystrophy. National Human Genome Research Institute. About Duchenne muscular dystrophy.
FierceBiotech7d
Solid Bio's stock soars as early DMD gene therapy data beat expectations
Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has ...
Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003
Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...
Managed Healthcare Executive13d
Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
Nanowerk8h
Using nanoparticles to transport microRNAs against Muscular Dystrophy to muscle stem cells
The scientists demonstrate that it is possible to specifically deliver the treatment to the muscle stem cells, avoiding ...
Solid Biosciences Stock Surges On Upbeat Duchenne Muscular Dystrophy Gene Therapy Data
Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.