[title]
Podcast: What Happened with Dexpramipexole for ALS?
January 15, 2013
[title]
MTM Mice Respond to Modified Myotubularin Protein Injections
January 10, 2013
Mice missing the muscle protein myotubularin and showing a disorder mimicking human myotubular myopathy (MTM) showed improvements in muscle structure and function after two weeks of treatment with a laboratory-modified form of the missing protein.
[title]
ALS Stem Cell Trial in Israel Is ‘Accelerated’
January 09, 2013
The Israeli Ministry of Health has given BrainStorm Cell Therapeutics approval to accelerate its current phase 1-2 safety trial of the company’s NurOwn stem cell therapy in amyotrophic lateral sclerosis (ALS).
[title]
Pfizer and Repligen to Collaborate on SMA Therapy Development
January 04, 2013
The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA)
[title]
Phase 3 Trial of Dexpramipexole in ALS Fails to Show Benefit
January 02, 2013
Biotechnology company Biogen Idec today announced disappointing results from its phase 3 trial of dexpramipexole, an experimental drug the company has been developing to treat amyotrophic lateral sclerosis (ALS).
[title]
DMD, BMD: Nationwide Children's Podcast Explores Dystrophin's Interactions
December 31, 2012
In a December 2012 podcast from Nationwide Children's Hospital, cell biologist Federica Montanaro discusses her team's recent progress in understanding how various proteins interact with dystrophin and how these interactions differ in
[title]
MD Briefs: Gene Therapy, Exon Skipping, Stem Cells
December 27, 2012
Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.
[title]
MD Briefs: Gene Therapy, Exon Skipping, Stem Cells
December 26, 2012
Update (Jan. 23, 2013):The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.
[title]
Federal SMA Project Issues Progress Report
December 24, 2012
A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (SMA) Project, a program established in 2003 by the
[title]
UPDATED: WNT7a Injections Restore Strength in DMD Mice
December 20, 2012
Injecting a muscle-repair protein known as WNT7a into the muscles of mice lacking the dystrophin protein and showing a disease resembling Duchenne muscular dystrophy (DMD) signif
[title]
ALS Briefs from the 2012 ALS/MND Symposium
December 18, 2012
The 23rd International Symposium on ALS/MND (motor neuron disease), held in Chicago Dec. 5-7, 2012, brought together more than 900 researchers, clinicians and other health care professionals from 30 countries to hear presentations on the latest in ALS care and research.
[title]
Second Mouse Model of Periodic Paralysis Developed
December 17, 2012
Scientists supported in part by MDA have developed a second type of research mouse with a disorder mimicking hypokalemic periodic paralysis, a genetic disorder in which recurrent attacks of weakness or paralysis occur in association with low potassium levels in the blo
[title]
ALS Briefs: Clinical Trial Updates at the 2012 ALS/MND Symposium
December 13, 2012
The 23rd International Symposium on ALS/MND (motor neuron disease), held in Chicago Dec. 5-7, 2012, brought together more than 900 researchers, clinicians and other health care professionals from 30 countries to hear presentations on the latest in ALS care and research.
[title]
Tirasemtiv Shows Promise in MG
December 12, 2012
The experimental drug tirasemtiv has shown promise in a phase 2a clinical trial in 32 people with myasthenia gravis (MG). Improvements in general and respiratory muscle function occurred in response to the medication.
[title]
What’s Next for Eteplirsen? Sarepta Discusses FDA Approval and Future Trials
December 07, 2012
In both October and December 2012, Sarepta Therapeutics announced very encouraging results from a 12-person phase 2b trial of eteplirsen, an exon-skipping compound that is a potential treatment for Duchenne muscular dystroph
[title]
DMD: Eteplirsen Results Still Strong at 62 Weeks
December 07, 2012
Eteplirsen, an exon-skipping drug in development by Sarepta Therapeutics to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show benefit at 62 w
[title]
MDA Building ‘Transitional Freedom’ Partnerships
December 06, 2012
[title]
European Agency to Consider Conditional Approval of Ataluren for DMD
December 05, 2012
New Jersey biopharmaceutical company PTC Therapeutics has announced that the European Medicines Agency (EMA) has validated a "marketing authorization application" (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of
[title]
ALS Research Briefs: Protein Biomarker and Stem Cells
December 04, 2012
Protein test predicts ALS progression
[title]
DMD: Sarepta Expands Exon-Skipping Program
December 04, 2012
Biopharmaceutical company Sarepta Therapeutics has announced it will expand the focus of its exon-skipping program for Duchenne muscular dystrophy (DMD) by developing compounds that target exons 45, 50 and 53 of the
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