Quest Magazine

Antisense Against C9ORF72 ALS and FTD

Results from three recent studies — two supported in part by MDA — shed light on how the chromosome 9 open reading frame 72 (C9ORF72) mutation causes nerve cell death in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

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

Prosensa, GSK Conducting DMD Natural History Study

MDA Funds Development of Drug to Fight Inflammation, Scarring in DMD

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

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