On Dec. 23, 2016 the FDA approved Spinraza for the treatment of SMA. In clinical trials to test Spinraza, participants who received treatment with the drug experienced life-changing outcomes they wouldn’t have been expected to achieve.
Ionis Pharmaceuticals provided an update on its recently-concluded phase 1-2 clinical trial of IONIS-DMPK-2.5Rx, an experimental compound for the treatment of type 1 myotonic dystrophy (DM1). Analysis showed mixed results from the trial.
Five Questions with Researcher Matthew Disney Matthew Disney, a professor at the Scripps Research Institute in Jupiter, Fla., was awarded an MDA research grant totaling $300,000 over a period of three years to optimize two novel drug-like compounds — one that targets toxic RNA in myotonic dystrophy and another that targets toxic RNA in ALS . . .
Researchers at the University of Kansas and Arkansas Children’s Hospital are looking for boys and men age 10 years and older with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial. The study, called SIDEROS, is designed to help researchers determine the safety and efficacy of idebenone (brand name Raxone), an experimental . . .
Researchers are seeking volunteers to participate in a clinical trial sponsored by ReveraGen BioPharma to test the safety and effectiveness of the investigational drug vamorolone in boys with Duchenne muscular dystrophy (DMD). In the two-part, open-label study called “A Phase 2a and 2a Extension study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy,” all . . .