clinical trial

CMD: Phase 1 Omigapil Trial Remains Open

Santhera Pharmaceuticals continues to recruit participants for a phase 1 trial of omigapil in children and adolescents with the type 1A form of congenital muscular dystrophy (CMD)

FDA OKs Testing of OPMD Drug in US

BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD).

ALS: Seven Out of 15 Participants in Neuralstem Trial Judged Responders

Maryland-based biotechnology company Neuralstem announced in a March 12, 2014, press release, that a phase 2 trial of its NSI-566 neural stem cells in 15 people with amyotrophic lateral sclerosis (ALS) has shown that the surgical transplantation procedure and the cells

SMA: ISIS-SMNRx To Be Tested in Presymptomatic Infants

Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx  in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms. 

NIH Will Test Antioxidant Drug in Central Core Disease

The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as ryanodine receptor 1 (RYR1).

Drug Development for DMD: February 2015 Update

Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene.

Fighting fibrosis

Genervon's 'Master Regulator' Drug Encourages ALS Community

Biopharmaceutical company Genervon has announced what it considers encouraging  results from a "compassionate use" trial of its experimental drug GM604 in a single patient with advanced  amyotrophic lateral sclerosis (ALS).

DMD: Eteplirsen Data Still Encouraging After Three Years

Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of eteplirsen.

Myostatin Blocker To Be Tested in DMD

Multinational pharmaceutical comany Pfizer recently opened a phase 2 study of an experimental compound that may be beneficial in Duchenne muscular dystrophy (DMD) through its ability to block myostatin, a naturally occurring protein that is known to limit muscle growth.

Sarepta Reaffirms Commitment to Eteplirsen, Other DMD Drugs

Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed  to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.

MDA in Your Community

Grants