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

Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene ...

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

Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed with Duchenne muscular dystrophy in 2010 at 4 years old. The other boys were diagnosed with the same in the ...

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 functions of full-length dystrophin and allows it be delivered more effectively ...

The cuts hit the FDA’s medical device center particularly hard, impacting product reviewers and researchers of AI-enabled ...

Boston, USA-based biotech Solid Biosciences saw its shares rocket 36% to $5.48 yesterday, after the precision genetic ...

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

Solid Biosciences Inc. (SLDB) shares surged more than 31% on Tuesday, their best session since January 2024, and extended ...