Solid Biosciences' SGT-003 gene therapy showed strong dystrophin expression, muscle health improvements, and no serious safety concerns in early trial data.

Solid Biosciences has surged back into the Duchenne muscular dystrophy (DMD) gene therapy race. | Solid Biosciences has ...

Solid Biosciences (SLDB) announced initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...

Brandon Looney, who grew up in the Ozarks, is bringing his benefit show to the Landers for one night as he raises money for ...

Understanding the ins and outs of a rare disease like atypical hemolytic uremic syndrome (aHUS) is a difficult task for ...

Despite the discontinuations, CEO Pascal Soriot said AstraZeneca’s December 2020 acquisition of Alexion was a “fantastic” ...

including anti-C5 antibody * ADAMTS13 deficiency (\<10% activity or \<0.1U/ml), and/or Shiga toxin-related hemolytic uremic syndrome (STx-HUS), and/or Positive direct Coombs test * Identified drug ...

Atypical Hemolytic Uremic Syndrome Market Forecast DelveInsight’s Atypical Hemolytic Uremic Syndrome Market report offers an in-depth understanding of the epidemiology and market trends in the 7MM. LA ...

The central pathogenic feature of thrombotic thrombocytopenic purpura/hemolytic uremic syndrome (TTP/HUS) is the formation of platelet aggregates, perhaps by damaged endothelial cells. The ...

They are thus reported to be useful for the treatment of age-related macular degeneration, schizophrenia, atypical hemolytic uremic syndrome, membranous nephropathy, myasthenia gravis, paroxysmal ...