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 ...
Nanowerk6h
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 ...
Muscular Dystrophy News7h
ENTR-601-44 trial for DMD adults amenable to exon 44 skipping OK’d
The FDA has lifted its hold and has given Entrada Therapeutics permission to launch a Phase 1b clinical trial to test ...
GlobalData on MSN7h
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 ...
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 ...
Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44
Receives FDA authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of ENTR-601-44 in adults living with ...
Pharma giant's pullback from gene therapy puts spotlight on North Carolina operations
One of the largest pharmaceutical companies in North Carolina is discontinuing a treatment produced in the state.
BioPharma Dive4d
Pfizer stops selling hemophilia gene therapy, citing weak demand
Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for ...
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. | ...
Massachusetts families, lawmakers fighting to keep NIH funding for life-saving medical research
Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.
News Medical5d
The Latest Advances in Cell and Gene Therapy
The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through ...
Hosted on MSN5d
What To Know About Duchenne Muscular Dystrophy (DMD)
Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...