I'm fighting a fatal disease that took the life of my brother. I work in biotech and know there are medical advances that ...

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

At UMass Chan Medical School, researchers, doctors, patients and biomedical executives voiced their concerns over cuts to NIH ...

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 ...

Wheelchairs 4 Kids, a nonprofit organization dedicated to improving the lives of children with mobility challenges, is ...

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

The company, now based in North Beach, has lost more than $2 billion over 29 years. Its only approved drug, called roxadustat ...

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 ...

Don Pettit has been at it again. Not content with dazzling earthlings with incredible photos captured from his current home aboard the International Space Station (ISS), the NASA astronaut has now ...

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 ...