Researchers are looking for individuals with SMA type 2 to participate in the phase 1 STRONG clinical trial, to test safety, dosing and proof of concept for efficacy for its SMA gene replacement therapy AVXS-101.
Andrew Lieberman of the University of Michigan Medical School was awarded an MDA research grant to test a modified antisense oligonucleotide (ASO) therapy to treat spinal-bulbar muscular atrophy.
Auinash Kalsotra of the University of Illinois was awarded an MDA research grant totaling $300,000 over three years to shed light on how defects develop in the heart in DM1.
Dean Burkin was awarded an MDA research grant to test the effects of an existing FDA-approved drug on the function of heart and skeletal muscle in a mouse model of DMD.
Angela Lek, a postdoctoral research fellow at Boston Children sHospital, was awarded an MDA development grant totaling $180,000 over three years to use cutting-edge techniques and a novel approach to search for drug targets in FSHD.
Feng Yue, of Purdue University was awarded an MDA development grant totaling more than $175,000 over three years to evaluate the therapeutic potential of a protein called PTEN in DMD.
Researchers are looking for people with MG to participate in a phase 2 clinical trial, to test the experimental drug RA101495 in people with generalized MG who are positive for acetylcholine receptor autoantibodies.
James Lupski, who lives with Charcot-Marie-Tooth disease type 4B., was awarded an MDA research grant totaling $300,000.
You’ve heard the term “biomarker,” but what exactly is a biomarker and why does it matter in regard to neuromuscular disease? Learn more here:
Solid Biosciences announced the launch of its first clinical trial for SGT-001. The phase 1/2 study, called IGNITE DMD, is designed to assess safety and efficacy of a single dose of SGT-001 in children and adolescents with DMD.
