SMA: Further data on ISIS-SMNRx shows promising results

SMA: Further data on ISIS-SMNRx shows promising results

Isis Pharmaceuticals updated the SMA community on phase 2 clinical trial results of the drug ISIS-SMNRx.

Phase 3 Clinical Trial for Eteplirsen Now Enrolling Boys with Duchenne Muscular Dystrophy

Phase 3 Clinical Trial for Eteplirsen Now Enrolling Boys with Duchenne Muscular Dystrophy

Boys between the ages of seven and 16 with Duchenne muscular dystrophy are being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.

LEMS: Firdapse Study Shows Encouraging Results

LEMS: Firdapse Study Shows Encouraging Results

Catalyst Pharmaceuticals launches Firdapse expanded access program for eligible people living with congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome

DMD: Sarepta Updates Community on Eteplirsen, Other Compounds

DMD: Sarepta Updates Community on Eteplirsen, Other Compounds

Sarepta Therapeutics updated the Duchenne MD community on regulatory and clinical trial progress with respect to eteplirsen, SRP-053 and SRP-045 on Aug. 7, 2014

MG: Multicenter Trial of Rituximab Opens

MG: Multicenter Trial of Rituximab Opens

A phase 2 clinical trial of rituximab in myasthenia gravis has now opened at 15 U.S. centers under the auspices of NIH's NeuroNEXT

DMD: Ataluren Receives Conditional Approval in Europe

DMD: Ataluren Receives Conditional Approval in Europe

Duchenne patients in the European Union with specific dystrophin gene flaws can receive the drug ataluren prior to its full approval

FEATURED IN QUEST MAGAZINE

News and updates from the MDA community

posted on July 7, 2014 - 9:11am
A Lasting Legacy: A family honors lost loved ones with a planned gift to MDA

Four professionals recently talked with Margaret Wahl, MDA’s medical and science editor, sharing their thoughts about access to drugs that have not gone through the full approval process

posted on July 7, 2014 - 9:09am
Can We Have It Now?

Drug development consultant Abby Bronson says early access to a new drug can jeopardize its full approval and long-term availability

posted on July 7, 2014 - 9:09am
Abby Bronson is the Duchenne muscular dystrophy program manager at Children's National Medical Center in Washington, D.C., where she works with various stakeholders to further therapeutic development for this disease. Bronson has a master's degree in business administration from the University of Pennsylvania and has managed new product development and marketing at biotechnology and...

Bioethicist Arthur Caplan says early access to drugs should not be determined by drug companies but by independent advisory boards

posted on July 7, 2014 - 9:09am
Arthur Caplan is the William F. and Virginia Connolly Mitty Professor and founding head of the Division of Bioethics at New York University Langone Medical Center in New York City. He holds a doctoral degree in the history and philosophy of science from Columbia University, has authored or edited 32 books and more than 600 papers, and has served on national and international committees as an...

FDA staffer Richard Klein says people often underestimate the risks and overestimate the benefits of taking medications that have not been through the full FDA approval process

posted on July 7, 2014 - 9:09am
Richard Klein is the director of the Patient Liaison Program at the Office of Health and Constituent Affairs at the U.S. Food and Drug Administration (FDA). Q: What is the most common misconception that patient communities have about the FDA's accelerated approval mechanism?

Neuromuscular disease specialist and biotechnology company employee Timothy Miller says it can be a significant risk and financial hardship for a small company to supply a drug that has not been through the full approval process

posted on July 7, 2014 - 9:09am
Timothy Miller has been a university-associated neurologist specializing in neuromuscular disorders who has recently moved to the biotechnology industry. He has been the director of the MDA-supported pediatric neuromuscular disorders clinic at Children's Clinics for Rehabilitative Services in Tucson, Ariz.; an assistant professor of neurology, pathology and pediatrics at the University of Arizona...