Business Insider17d
AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort
Adult participants diagnosed with LGMD2I ... infusion. 2-4 About Limb-Girdle Muscular Dystrophy Type 2I/R9 LGMD2I/R9 is a rare form of LGMD caused by mutations in the FKRP gene and is associated ...
University of Bristol2y
GEM Study
to take part in a research study which will tell us whether late-onset Pompe disease or Limb Girdle Muscular Dystrophy 2A is found in children, young people and adults with ME/CFS. Pompe disease (also ...
Morningstar17d
AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort
Adult participants diagnosed with LGMD2I ... investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe ...
Yahoo Finance17d
AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort
today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9 ...
Financial Post18d
AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort
1 The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants ... heart failure ...