Quest Magazine

CoQ10, Lisinopril Trial in DMD, BMD, LGMD2C-2F, LGMD2I

Researchers at five U.S. and one Canadian center are conducting a clinical trial of the medications coenzyme Q10 and lisinopril to determine their possible beneficial effects on heart function in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and five forms of limb-girdle muscular dystrophy (LGMD).

ALS Clinical Trials Briefs: Cytokinetics, Neuralstem Advance

Editor's note 3/19/12: This story has been updated to reflect that Neuralstem has begun testing its stem cells in the cervical (neck) region of the spinal cord.

Two human clinical trials to test therapies in amyotrophic lateral sclerosis (ALS) are moving forward.

Researchers Studying CMT1B, CMT2A, CMT4A, CMT4C, Others

A large-scale study, supported in part by MDA, seeks to determine the natural history (general disease course) of four subtypes of Charcot-Marie-Tooth disease (CMT), with particular emphasis on correlations between genetic mutations and symptoms. The four subtypes are CMT1B, CMT2A, CMT4A and CMT4C.

Albuterol Improved Quality of Life in Two Forms of CMS

Researchers have found that the drug albuterol appears to be beneficial in two forms of congenital myasthenic syndrome (CMS)— CMS related to mutations in the collagen Q (colQ) gene and CMS related to mutations in the DOK7 gene.

BioMarin Testing Drug in Late-Onset Pompe Disease

Update (March 27, 2013): BioMarin announced March 19, 2013, that it plans to move to a phase 2/3 trial of this drug for late-onset Pompe diseae, pending a review by regulatory authorities. See Pompe disease: Late-onset treatment moves forward.

Myasthenia Gravis Trials Now Open

Two clinical trials — one of an experimental medication and the other of a surgical procedure —  are available to people with myasthenia gravis (MG) who meet enrollment criteria.

CK-2017357 being tested at multiple centers

‘Aberrant Astrocytes’ Kill Motor Neurons in Rats

A multinational team of scientists has identified a specific type of astrocyte that behaves abnormally, causing degeneration and death in motor neurons in rats with a disease resembling familial SOD1-related ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

The astrocyctes have been named “aberrant astrocytes,” or AbA cells, by the research team, which has members from several institutions in Montevideo, Uruguay, and Oregon State University in Corvallis, Ore.

CMT Genetics Study Seeks Participants

Researchers supported in part by MDA are seeking people with Charcot-Marie-Tooth disease (CMT) to participate in a study to identify genetic modifiers of the type 1A form of CMT (CMT1A) and determine previously unknown genetic causes of CMT.

More Evidence for Astrocyte Involvement in ALS

An MDA-supported team of scientists has demonstrated in rats that nervous system support cells called astrocytes carrying an SOD1 mutation cause neighboring motor neurons to deteriorate and die.

The finding adds to a growing body of evidence that the star-shaped cells play a key role in the motor neuron degeneration that is the hallmark of ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease). Astrocytes normally support and protect motor neurons.

NIAMS Establishes MD Research Center

A Center for Research Translation of Systemic Exon Skipping in Muscular Dystrophy has been established by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the U.S. National Institutes of Health.