Five Questions with ALS Researcher Udai Pandey
Udai Pandey was awarded an MDA research grant totaling $300,000 over three years to identify new drugs for ALS caused by a mutation in the FUS gene.
Author: Amy Madsen
Disappointing Results for Tirasemtiv in VITALITY-ALS Trial
Cytokinetics has reported negative results from its international phase 3 VITALITY-ALS trial to test the investigational drug tirasemtiv in people with ALS.
FDA Grants Orphan Drug Designation to Jupiter Orphan Therapeutics’ Experimental Friedreich’s Ataxia Drug JOTROL
The investigational drug JOTROL under development by Jupiter Orphan Therapeutics to treat FA has received FDA Orphan Drug Designation.
Trial to Test Drug Targeting Muscle Cramps Seeks Participants
Researchers are looking for people with MNDs like ALS to join a clinical trial for the investigational drug FLX-787-ODT.
Participants Sought for FORTITUDE ALS Trial
Researchers are looking for people with ALS to participate in a phase 2 clinical trial for the investigational drug CK-2127107.
Participants Sought for FIREFISH SMA Study
Researchers are looking for individuals with type 1 SMA for a clinical trial to test the investigational drug RG7916.
Participants Sought for SMA Gene Therapy Study
Researchers are looking for individuals with type 1 SMA to test efficacy for its SMA gene replacement therapy AVXS-101.
MDA Grant Will Support Development of ALS Biomarkers as a Predictive Test
MDA has awarded a grant to Iron Horse Diagnostics to support development of a prognostic (predictive) test for ALS.
PTC Therapeutics: Letter to the DMD Community
PTC Therapeutics President and CEO Stuart Peltz, Ph.D., issued a letter to the DMD community after the FDA declined to approve ataluren (brand name Translarna) for the treatment of DMD.
FDA: Translarna is Not Ready for Approval at This Time
PTC Therapeutics reported today the U.S. Food and Drug Administration’s Office of Drug Evaluation is unable to approve the company’s New Drug Application for ataluren (brand name Translarna) for the treatment of Duchenne muscular dystrophy (DMD) caused by a premature stop codon, or nonsense mutation, in its current form.
