Targeting multiple pathways in mice with a disorder resembling merosin-deficient congenital muscular dystrophy shows more promise than aiming at one pathway at a time

posted on July 11, 2013 - 11:15am
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.

Treatment with IGF1 improved motor function, slowed weight loss, improved muscle health and increased survival time in mice with a disease resembling spinal-bulbar muscular atrophy

posted on September 10, 2012 - 5:00am
Mice with a disease resembling spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) that were treated with a compound based on insulin-like growth factor 1 (IGF1) had better motor function, slower weight loss, healthier muscles and longer survival time than mice that received an inactive substance, an MDA-supported research team has reported.
posted on October 1, 2009 - 1:53pm
QUEST Vol. 16, No. 4
The following article contains items about: Friedreich's ataxia, Charcot-Marie-Tooth disease, myotonic muscular dystrophy type 1, amyotrophic lateral sclerosis, Emery-Dreifuss muscular dystrophy and distal muscular dystrophy (Miyoshi myopathy)

Insmed to evaluate potential initiation of phase II trial for IPLEX
in MMD patients with severe insulin resistance

posted on June 25, 2009 - 7:08am
RICHMOND, VA., June 25, 2009 - Insmed Inc. (NASDAQ CM: INSM), a biopharmaceutical company, today announced results from its exploratory U.S. Phase II clinical trial evaluating IPLEX™ (mecasermin rinfabate) in patients with myotonic muscular dystrophy (“MMD”).  The randomized, double-blind, placebo-controlled Phase II trial conducted in 13 centers across the U.S.
posted on May 1, 2008 - 10:21am
QUEST Vol. 15, No. 3
This article includes items about: Duchenne muscular dystrophy, limb-girdle muscular dystrophy, Becker muscular dystrophy, myotonic muscular dystrophy, fascioscapulohumeral muscular dystrophy, Charcot-Marie-Tooth disease, spinal muscular atrophy, Friedreich's ataxia, myasthenia gravis and centronuclear myopathy
posted on September 1, 2007 - 11:06am
QUEST Vol. 14, No. 5
Featured in this issue: News about clinical trials in Charcot-Marie-Tooth disease, Duchenne muscular dystrophy, Pompe disease and myotonic muscular dystrophy type 1