Could MRI be better than muscle biopsy in measuring disease progression and therapy effectiveness in Duchenne muscular dystrophy?
posted on May 17, 2010 - 1:20pm
The use of magnetic resonance imaging (MRI) as an assessment tool in boys with Duchenne muscular dystrophy (DMD) is being studied by former MDA grantee Krista Vandenborne, through a $7.5 million grant from the National Institutes of Health (NIH).
The goal of the study is to assess whether MRI technology can be used as a precise, noninvasive measure of muscle tissue, gauging both disease...
A variant in the gene for the osteopontin protein has the potential to help predict disease course in Duchenne MD
posted on January 11, 2011 - 2:58pm
A team of scientists working in the United States and Italy has uncovered a variant in the gene for a protein called osteopontin that appears to reliably indicate disease severity in most (but likely not all) cases of Duchenne muscular dystrophy (DMD).
The variant is apparently a genetic modifier of DMD, a disease in which the underlying cause is a mutation in the gene for the dystrophin protein...
Repligen soon will begin a first-ever human trial of a drug created specifically for spinal muscular atrophy; FDA grants fast track designation
posted on May 20, 2011 - 11:13am
In a historic first, biotech company Repligen Corp., of Waltham, Mass., has received approval from the U.S. Food and Drug Administration (FDA) to begin a phase 1 clinical trial of the experimental drug RG3039 for spinal muscular atrophy (SMA).
An MDA-supported open-label trial found the asthma drug benefited 17 of 18 people with either of two forms of congenital myasthenic syndrome
posted on November 9, 2011 - 2:56pm
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.
Top researchers are discussing notable developments in neuroscience, including research in many neuromuscular diseases, at the annual American Academy of Neurology meeting April 21-28
posted on April 24, 2012 - 4:00am
Top scientists and clinicians from around the world are discussing the latest research in neuroscience, and the care of individuals with nerve and muscle diseases, at the 2012 annual meeting of the American Academy of Neurology in New Orleans, April 21-28.
Findings about ANO5 mutations, DNAJB6 mutations and dysferlin gene transfer were presented
posted on May 9, 2012 - 12:15pm
The 2012 annual meeting of the American Academy of Neurology (AAN), held in New Orleans April 21-28, included findings related to limb-girdle muscular dystrophy (LGMD) and distal muscular dystrophy.
After a two-year review, the FDA approves reopening a clinical trial of the experimental compound arimoclomol.
posted on December 8, 2009 - 5:45pm
A clinical trial of an experimental drug for ALS -- halted almost two years ago due to safety concerns –- has been given the green light to continue with a revised protocol, says CytRx Corp. of Los Angeles, the drug’s manufacturer.
The U.S. Food and Drug Administration placed a hold on the phase 2b trial of arimoclomol in January 2008, saying it wanted to see more toxicity data from previously...
Early results show that when AVI4658 is delivered system-wide through the bloodstreams of boys with DMD, it’s safe and increases dystrophin production.
posted on December 23, 2009 - 3:07pm
Interim results from a human clinical trial of the exon-skipping compound AVI4658 in boys with Duchenne muscular dystrophy (DMD) show that when the compound is delivered to the whole body via the bloodstream — rather than simply injected into a foot muscle as in a previous trial — it appears safe and leads to production of the missing muscle protein dystrophin.
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.
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).
