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 ...
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.
Nanowerk4h
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 ...
GlobalData on MSN6d
Solid Biosciences’ stock soars on early success in DMD gene therapy trial
The trial was able to elicit more than double the microdystrophin gene in patients after 90 days of treatment.
FiercePharma4d
Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...
Managed Healthcare Executive6d
Solid Biosciences Reports Positive Data from DMD Gene Therapy Trial
Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively ...
FDA Clears Entrada Therapeutics' Early-Stage For Potential Treatment Of Duchenne Muscular Dystrophy
Entrada Therapeutics gains FDA clearance for its Phase 1b ELEVATE-44-102 study, evaluating ENTR-601-44 in adult Duchenne ...
pharmaphorum1d
Sarepta files Duchenne muscular dystrophy gene therapy with FDA
Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...
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 ...
pharmaphorum1d
Pfizer drops Beqvez, leaving its gene therapy cupboard bare
Beqvez was Pfizer's first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) more than a ...
The Pharma Letter6d
Solid Biosciences surges on Duchenne therapy success
Boston, USA-based biotech Solid Biosciences saw its shares rocket 36% to $5.48 yesterday, after the precision genetic ...
Taiwan News6d
Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003
Average microdystrophin expression of 110% (N=3) and significant improvements in multiple additional muscle health biomarkers observed support the potential of SGT-003 as a next-generation, ...