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

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

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 innovative gene editing approaches.

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 ...

Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key ...

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

Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.

Solid Biosciences shares were up 50% to $6.06 after the company said it saw positive initial data from a Phase 1/2 trial evaluating SGT-003, a gene therapy product candidate intended for the treatment ...

Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin expression of 110% ...

Caleb Kasner and his brother Duncan Kasner move around a little differently than average kids. The two brothers have Duchenne ...