Drugs

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

posted on August 11, 2014 - 10:15am
Cambridge, Mass.-based biotechnology company Sarepta Therapeutics updated the Duchenne muscular dystrophy (DMD) community about its pipeline of drugs in development for this disorder via an Aug.

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

posted on August 11, 2014 - 8:18am
A phase 2 clinical trial of the drug rituximab (Rituxan) in adults with myasthenia gravis (MG) is now open at 15 U.S. centers with plans for additional sites. Rituximab suppresses a specific part of the body's immune system and is approved by the U.S. Food and Drug Administration (FDA) to treat rheumatoid arthritis and other disorders.

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

posted on August 4, 2014 - 2:44pm
The investigational oral drug ataluren, in development to treat Duchenne muscular dystrophy (DMD) resulting from a specific type of genetic mutation, has received conditional approval in the European Union (EU). This designation means patients can receive it in the EU and EU-associated countries while additional studies are being conducted.

Isis Pharmaceuticals is testing its experimental, antisense-based drug in a phase 3 trial in SMA-affected babies age 7 months or less

posted on August 1, 2014 - 10:24am
California-based Isis Pharmaceuticals has announced the opening of a phase 3 clinical trial of its experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA), a muscle-weakening disease that results from loss of nerve cells in the spinal cord.

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
Quest Summer 2014
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
Quest Summer 2014
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
Quest Summer 2014
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
Quest Summer 2014
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...