posted on January 31, 2009 - 10:00pm
On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom.
posted on November 1, 2006 - 2:24pm
This article contains news of clinical trials in: myasthenia gravis, limb-girdle muscular dystrophy, polymyositis, dermatomyositis, inclusion-body myositis, and unapparent forms of muscular dystrophy
Different mutations in a gene called ANO5 cause type 2L LGMD (LGMD2L) and type 3 Miyoshi myopathy
posted on February 18, 2010 - 1:44pm
An MDA-supported, multinational team of researchers from Canada and Europe has identified specific mutations in the anoctamin 5 (ANO5) gene on chromosome 11 that can cause type 2L limb-girdle muscular dystrophy (LGMD2L) and type 3 Miyoshi myopathy.
posted on March 31, 2010 - 2:56pm
Featured in this issue: Disappointing results from ataluren trial leads to further analysis * First U.S. site for idebenone in DMD trial opens * Utrophin-boosting drug begins safety tests * Participants sought for trials in DMD, CMD, myotonic distrophy and periodic paralysis
The vasodilating drug tadalafil (Cialis) will be tested in men with Becker muscular dystrophy to see if it improves blood flow to forearm muscles
posted on March 24, 2010 - 9:15am
The oral drug tadalafil (brand name Cialis), commonly used to treat erectile dysfunction, is being tested in an MDA-supported trial to see whether it can improve blood flow to forearm muscles in adults with Becker muscular dystrophy (BMD).
Further analysis of data from a trial of the exon-skipping drug AVI4658 showed some participants had greatly increased dystrophin production
posted on June 2, 2010 - 10:15am
The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD).
The new results show that, at higher doses, AVI4658 can result in substantial production of the needed dystrophin protein in muscle fibers.
The company has not yet released results of any tests of muscle...
posted on October 22, 2009 - 3:47pm
A technique called exon skipping shows great potential to increase muscle strength and prolong life in people with a severe form of Duchenne muscular dystrophy (DMD). According to a study that included MDA-supported Stephen Wilton at the University of Western Australia, exon skipping improves production of a crucial muscle protein that’s missing in people with DMD. For the first time, these...
The first ALS trial to block abnormal SOD1 protein using ‘antisense’ is set to open by the end of 2009
posted on October 15, 2009 - 5:00pm
A phase 1 clinical trial of the experimental drug ISIS-SOD1-Rx in patients with the SOD1-related type of familial (inherited) amyotrophic lateral sclerosis (ALS) is expected to begin before the end of 2009 at Washington University in St. Louis, Massachusetts General Hospital in Boston and four additional U.S. sites.
A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake
posted on September 28, 2009 - 5:00pm
Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experimental compound being developed to treat this disease. MDA is supporting Repligen to develop this...
A variant in the SMN2 gene leads to more full-length SMN protein and better function
posted on September 24, 2009 - 5:00pm
Scientists have uncovered a variant (mutation) in the SMN2 gene that leads to production of more full-length SMN protein molecules and a milder version of spinal muscular atrophy (SMA). The finding, a naturally occurring point mutation (a single letter change in the DNA code) in this gene, has immediate implications for genetic testing and possible long-term implications for therapy development.
