Quest Magazine

Nationwide Children’s Podcast Explores Therapy Development for DMD

In a July 2013 podcast from Nationwide Children’s Hospital in Columbus, Ohio, longtime MDA grantee Jeffrey Chamberlain discusses recent advances in the development of gene therapy (gene transfer) and stem cell therapy (transplantation) for Duchenne muscular dystrophy (DMD), the results of which may apply to other types of muscular dy

DMD: Multicenter Trial To Test Drug that Fights Muscle Scarring, Inflammation

A phase 1b/2a clinical trial to test the safety, tolerability and pharmacokinetics of single and multiple doses of HT-100 (also called delayed-release halofuginone) in boys with Duchenne muscular dystrophy (DMD) is now open at four sites in Maryland, Missouri and Ohio, with an additional site expected to open in California.

MDA Statement RE: Sarepta Therapeutics To Submit NDA for Duchenne Muscular Dystrophy Next Year

'Sugar' Molecule Delays Disease Progression in ALS Mice

Treatment with a small molecule called trehalose was associated with slower disease progression and longer life span in mice with a disease mimicking amyotrophic lateral sclerosis (ALS), an MDA-supported research team has reported.

Trehalose is a compound composed of two glucose (sugar) molecules.

DMD: Sarepta to Submit New Drug Application to FDA for Eteplirsen in 2014

 In a July 24, 2013, press release and conference call, biotechnology company Sarepta Therapeutics provided an update on the regulatory pathway for eteplirsen, the company’s experimental drug for Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin

Study of Pregnancy in Women with Myotonias

Investigators at the University of Rochester (N.Y.) are seeking participants for a questionnaire-based study of pregnancy and delivery in women with nondystrophic myotonias. No travel is required.

Nondystrophic myotonias included in this study are:

Multicenter Trial to Test Safety of Mexiletine in Sporadic ALS

The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking participants for a phase 2 clinical trial that will evaluate the safety and tolerability of mexiletine in people with the sporadic form of amyotrophic lateral sclerosis (ALS).

Approximately 90 to 95 percent of ALS is sporadic, in which there is no known family history of the disease.

Friedreich's Ataxia Registry Open for Enrollment

The Coordination of Rare Diseases at Sanford (CoRDS) national rare disease registry is now hosting an ataxia patient registry for people with Friedreich's ataxia (FA) or other disorders classified as ataxias (conditions that cause problems with balance or coordination).

CMD: Aiming Simultaneously at Two Biological Targets

Researchers at Boston University, supported in part by MDA, say their experimental two-pronged strategy for merosin-deficient congenital muscular dystrophy (MDC1A) was highly successful in a mouse model of this disease and should be further investigated as a potential treatment approach for patients.

Turning Myotonic Dystrophy On and Off in Cells

MDA grantee Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).

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