New Biomarker for C9 ALS
An MDA supported project led to the discovery of a promising biomarker that could be used in developing and testing therapeutics to treat ALS.
Author: Amy Madsen
Five Questions with ALS Researcher Jie Jiang
Jie Jiang, postdoctoral fellow at the University of California – San Diego, was awarded an MDA development grant to improve understanding of ALS.
Everything We Know So Far about Access, Cost and Availability of DMD Drug Emflaza
Find answers to your questions about access, cost and availability of DMD drug Emflaza.
Simply Stated: What is ALS?
While many have heard of ALS, a much smaller group actually knows what causes it and who it impacts.
Five Questions with ALS Researcher Fen-Biao Gao
Dr. Fen-Biao Gao was awarded an MDA research grant to study the C9ORF72 repeat expansion, which is the most common genetic mutation known to cause ALS.
FDA Approves Radicava to Treat ALS
Today, on the first Friday of ALS Awareness Month, the U.S. Food and Drug Administration (FDA) approved edaravone (brand name Radicava), to treat ALS (amyotrophic lateral sclerosis). Under development by Mitsubishi Tanabe Pharma America, Radicava was approved in 2015 to treat ALS in Japan. It’s the first drug to be granted FDA approval to treat ALS in the United States in more than 20 years.
Five Questions with ALS Researcher Eric Shoubridge
Dr. Eric Shoubridge of McGill University in Montreal, Québec, who was awarded an MDA research grant totaling $287,169 over three years to study a new mitochondrial gene linked to ALS.
Promising results in phase 1 SMA gene therapy trial
Chicago-based biotechnology company AveXis has announced encouraging results from its completed phase 1 gene therapy trial to test the investigational therapy AVXS-101 in infants with type 1 SMA.
Encouraging Trial Data Reported for ALS Drug Masitinib
AB Science has reported encouraging top line trial results from its completed phase 2/3 study in Europe of the experimental drug masitinib in ALS. Additional testing is likely required, but if successful, masitinib could one day become an approved therapy for ALS.
Trial Seeks Participants to Test CK-2127107 in SMA
Investigators are seeking participants for an ongoing phase 2 clinical trial, sponsored by Cytokinetics, to test the investigational drug CK-2127107 in people with types 2, 3 or 4 SMA.
