Patients, their families and caregivers, researchers and clinicians are invited to gain insight into the testing process for experimental therapies to treat amyotrophic lateral sclerosis (ALS) via a live webcast this month. The ALS Clinical Trials Guidelines 2016 Workshop, an international effort that seeks to renew and update the current ALS Clinical Trials Guidelines, will . . .
Grace Hometown: Middletown, Delaware Age: 15 Diagnosis: I was diagnosed with myotonia congenita in 2006 at the MDA Clinic at A.I.duPont Hospital. Myotonia congenita is an inherited myopathy, a disease that causes problems with the tone and contraction of skeletal muscles. Favorite School Subject/Activities: Digital arts, music, and literature. Favorite People and/or Pets: I live with my parents, . . .
In two webcasts held Feb. 29 — one for investors and one for Duchenne muscular dystrophy (DMD) patients and families — PTC Therapeutics addressed the Refuse to File letter it received last week from the U.S. Food and Drug Administration (FDA) stating that PTC’s New Drug Application (NDA) for Translarna (ataluren) to treat DMD was not sufficient . . .
Researchers are looking for people with amyotrophic lateral sclerosis (ALS) to participate in a phase 1 clinical trial, sponsored by Biogen and Ionis Pharmaceuticals, to test the experimental drug BIIB067 (Ionis-SOD1Rx). BIIB067 is an antisense oligonucleotide, composed of short segments of synthetic genetic material (nucleic acid) that bind to RNA. The drug is designed to . . .
