Next Generation Exon Skipping Therapies Developed for the Treatment of Duchenne Muscular Dystrophy
Next generation exon skipping therapies for Duchenne Muscular Dystrophy (DMD) represent a promising area of research aimed at addressing the underlying genetic mutations that...
Insmed’s Gene Therapy (INS1201) Poised to Challenge Duchenne Muscular Dystrophy (DMD)
Insmed is getting ready to use its ground-breaking gene therapy, INS1201, to combat Duchenne muscular dystrophy. Insmed intends to start Phase I clinical trials...
Solid Biosciences Releases Information on Clinical Trial for SGT-003 Gene Therapy
Solid Biosciences shared that four patients have been dosed in the SGT-003 gene therapy clinical trial; it was well tolerated in all patients with...
Dr. Liubov Gushchina’s Exon 17 Skipping Study Rewarded with Grant
We are happy to present the winners of the American Neuromuscular Foundation's (ANF) 2024 Investigator Grant Award. Liubov Gushchina, PhD, received the prize for...
The FDA Designates IPS HEART’s GIVI-MPC Stem Cell Therapy for Becker Muscular Dystrophy an Orphan Drug
The FDA has granted IPS HEART Orphan Drug Designation (ODD) for GIVI-MPCs because of its exceptional capacity to produce new muscle with full length...