Children with a rare form of muscular dystrophy have been promised access to a groundbreaking drug after the Sunday Mail highlighted their plight. Around 30 boys could have their lives extended by ...

Children with a rare form of muscular dystrophy have been promised access to a groundbreaking drug after the Sunday Mail highlighted their plight. Around 30 boys could have their lives extended by ...

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy, the biotech has closed a public offering that raised an impressive ...

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD ... muscle wasting in the face, shoulder blades ...

Her son, Vicente Giron Sarria, had been diagnosed with facial deformities, and Lakatos began joining Blanco and her partner at every meeting with the craniofacial team. "They would explain [the ...

The parents of a little girl banned from nursery school - because officials said her skull deformity would scare other children - have faced further heartache after doctors said they can't help her.