Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared
Bru-nO / Pixabay

Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared

Sangamo Therapeutics has shared details of their preliminary data from a clinical trial of the investigational drug SB-525 in patients with Haemophilia A. For more detailed information, you can read…

Continue Reading Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared

Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…

Continue Reading Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

A Group of Disorders Share Similarities in Their DNA 3D Folding Patterns, Researchers Say

Researchers at the University of Pennsylvania have studied the genetics of a class of neurological disorders that includes Huntington’s disease and Fragile X Syndrome. They found that there were similarities…

Continue Reading A Group of Disorders Share Similarities in Their DNA 3D Folding Patterns, Researchers Say

A Boy With Congenital Muscular Dystrophy is Being Supported in a Special Way

Michael Dickerson, aged 42, is originally from Hoedspruit Limpopo, South Africa. Recently, Michael was participating in a series of long distance trail runs when he learned about the story of Daniël…

Continue Reading A Boy With Congenital Muscular Dystrophy is Being Supported in a Special Way
ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment
www_slon_pics / Pixabay

ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment

According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…

Continue Reading ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment