The scientists demonstrate that it is possible to specifically deliver the treatment to the muscle stem cells, avoiding ...

The FDA has lifted its hold and has given Entrada Therapeutics permission to launch a Phase 1b clinical trial to test ...

Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA ...

Receives FDA authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of ENTR-601-44 in adults living with ...

One of the largest pharmaceutical companies in North Carolina is discontinuing a treatment produced in the state.

Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...

Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.

The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through ...

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 ...

The trial was able to elicit more than double the microdystrophin gene in patients after 90 days of treatment.

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 ...