A surge of generics, biosimilars, and innovative therapies is expected in 2025, addressing conditions from cancer to chronic ...

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Catalyst Pharmaceuticals (CPRX) reported that its sub-licensee in Canada, Kye Pharmaceuticals, has announced that Health Canada has accepted ...

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

Kye Pharmaceuticals announced it has submitted a New Drug Submission, or NDS, to Health Canada for the regulatory review and approval of ...

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

Suneel Ram's caregivers share some of the lessons they have learned while caring for the 28-year-old who was diagnosed with ...

Researchers in pharma and beyond have historically glommed onto a limited number of disease targets, limiting innovation. AI ...

Suneel Ram is a spiritual seeker, sings in a band and was the subject of a comic novel two years ago about living with Duchenne muscular dystrophy, a rare genetic ...

Patients in Scotland can't get the treatment - which is available in England - despite manufacturers giving it away for free ...