clinical trial

MTM Natural History Study Seeks Participants

High-Calorie Diet May Help in ALS

A study supported in part by MDA has found that a high-calorie, high-carbohydrate diet is safe and well-tolerated in late-stage amyotrophic lateral sclerosis (ALS), that it leads to moderate weight gain, and that it may slow disease progression.

DMD: Prosensa to Present Webcasts on Drug Development

Dutch biopharmaceutical company Prosensa, developer of the experimental drug drisapersen and other experimental compounds for Duchenne muscular dystrophy (DMD), will update the investor community via a Thursday, Jan. 16, 2014, presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation will be webcast at 10 a.m. Pacific time/1 p.m. Eastern time.

On Tuesday, Jan. 21, at 8 a.m. Eastern time, Prosensa will conduct a separate webinar for DMD patients and families.

DMD: Eteplirsen-Treated Boys Show Continued Stability at 120 Weeks

Boys with Duchenne muscular dystrophy (DMD) participating in a phase 2b extension trial of the experimental exon-skipping drug eteplirsen showed continued stabilization of walking distance at 120 weeks. Sarepta Therapeutics, developer of eteplirsen, announced the findings in a Jan. 15, 2014, press release.

International ALS Symposium Stresses Progress

"We are undoubtedly slowing down the MND [motor neuron disease] supertanker, and can start to see how we might turn it around for the first tim," said Martin Turner, senior clinician scientist at the University of Oxford (United Kingdom), on research being reported on at the 24th International Symposium on ALS/MND.

ALS Nuedexta Study Seeks 60 Participants

The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking people with amyotrophic lateral sclerosis (ALS) to participate in a phase 2 clinical trial designed to evaluate whether Nuedexta has any effects on bulbar functions including speech, swallowing and saliva function.

Abnormal Immune Response to LRP4 Protein Can Cause MG

A research team supported in part by MDA has shown that an abnormal reaction of the immune system against a protein called LRP4 can be added to the known causes of myasthenia gravis (MG), a disorder involving fluctuating weakness and fatigue because of impaired nerve-to-muscle communication. MG is an autoimmune disease, meaning it's caused by abnormal activity of the body's immune system against its own tissues.

BMD, IBM: Outcome Measures Study Seeks Participants

A study to determine the best outcome measures — ways to evaluate the effects of a treatment — for use in clinical studies in Becker muscular dystrophy (BMD) and sporadic (nongenetic) inclusion-body myositis (sIBM) is underway at Nationwide Children's Hospital in Columbus, Ohio, under the supervision of neurologist Jerry Mendell working with physical therapists Linda Lowes and

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).

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).

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