FDA Grants Orphan Drug Designation to Octapharma USA’s Experimental Dermatomyositis Drug Octagam
Octapharma USA’s investigational drug IVIG (brand name Octagam) to treat dermatomyositis, has received FDA orphan drug designation.
Author: Amy Madsen
AMO Pharma’s Myotonic Dystrophy Drug AMO-02 Receives FDA Fast Track Designation
The investigational drug AMO-02 (tideglusib) has received FDA fast track designation for the treatment of DM1.
Five Questions with FSHD Researcher Charles Emerson
Dr. Charles Emerson was awarded an MDA research grant to study FSHD.
Five Questions with FSHD Researcher Scott Harper
MDA awarded a research grant to Scott Harper of Ohio State University to test a gene therapy for FSHD.
Five Questions with Researchers Michael Benatar and Jonathan Katz
Michael Benatar and Jonathan Katz were awarded a clinical research network grant from MDA to support their work on Clinical Procedures To Support Research (CAPTURE) project.
$300,000 MDA Grant Will Streamline Gathering of Data for ALS Research
MDA in June announced the award of a clinical research network grant (CRNG) to Michael Benatar, M.D., Ph.D., at and Jonathan Katz, M.D.
Advisory Committee Meeting Scheduled for Translarna
Translarna, a new drug under development that may slow functional decline in individuals with DMD cause by nonsense mutations, will be reviewed by an Advisory Committee of the U.S. Food and Drug Administration on Sept. 28, 2017. An FDA decision on the drug is expected on or before Oct. 24, 2017.
Simply Stated: What Causes ALS?
While there is little known about what causes ALS, scientists continue seeking out answers in the hope of finding new treatments and cures.
Simply Stated: What Are the Symptoms of ALS?
ALS is a disease of the parts of the nervous system that control voluntary muscle movement. In ALS, motor neurons are gradually lost and the muscles they control become weak and then nonfunctional.
Participants Sought for Answer ALS Study
Researchers are looking for people to participate in the Answer ALS: Individualized Initiative for ALS Discovery study.
