Quest Magazine

Halo Therapeutics Receives $500,000 MDA Award to Develop Drug for Duchenne Muscular Dystrophy

RE: FDA Says That New Drug Application Filing for Eteplirsen is Premature

FDA Says New Drug Application Filing for Eteplirsen is Premature

Utrophin Modulator SMT C1100 To Be Tested in DMD Patients

Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it has received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and Ethics Review Committee to begin testing its experimental drug SMT C1100 in children with Duchenne muscular dystrophy (DMD).

Multicenter Trial to Test Immunosuppression in ALS

Trial investigators are actively seeking people with amyotrophic lateral sclerosis (ALS) to participate in a phase 2 clinical trial that will evaluate the effects of a five-drug treatment regimen used to suppress immune system activity.

BMD, IBM: Preliminary Findings in Follistatin Gene Transfer Study Promising

Preliminary results from a trial to test the safety of injecting follistatin genes into the thigh muscles of adults with Becker muscular dystrophy (BMD)or sporadic (nongenetic) inclusion-body myositis (sIBM) suggest that the experimental therapeutic approach is safe in both types of patients

CNM/MTM Registry, World Map Seek Participants

A patient registry and world map of people with centronuclear myopathies (CNM), including myotubular myopathy (MTM), are being developed and are seeking participation from people with these disorders or their family members.

MDA Grant Will Help Families in DMD Steroid Study

Robert Griggs, a professor of neurology at the University of Rochester (N.Y.), has received an MDA grant of $237,316 over three years to support travel costs for North American participants in a large, multinational trial to determine which corticosteroid treatment regimen is best for children with Duchenne muscular dystrophy (DMD).

VA Biorepository Brain Bank Seeks Veterans with ALS

The U.S. Department of Veterans Affairs (VA) is seeking veterans who have amyotrophic lateral sclerosis (ALS) and who live in the United States to enroll in its VA Biorepository Brain Bank (VABBB), a human tissue bank that collects, processes and stores tissue samples for use in future research studies.

DMD: Sarepta Updates Community on Eteplirsen Plans, Pipeline

Sarepta Therapeutics announced it is on track to submit an application to the U.S. Food and Drug Administration (FDA) for approval of eteplirsen in the first half of 2014.

In parallel with that application, the company is preparing to conduct a large-scale, confirmatory trial of eteplirsen. It plans to open the trial during the first quarter of 2014.

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