Research News
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/Human_silhouette_blue.jpg)
May 15, 2012
The biotherapeutics company Neuralstem is seeking to amend the design of its ongoing phase 1 stem cell trial in amyotrophic lateral sclerosis (ALS) to include evaluations of efficacy (how well the
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/globe-man-laptop-info-arc%20copy.jpg)
May 15, 2012
Below are highlights of two recent studies in spinal muscular atrophy (SMA), a disease in which the nerve cells (motor neurons) that control muscles in the spinal cord die, causing progressive
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/couple-silhouette-moon.jpg)
May 11, 2012
Decision making about preimplantation genetic diagnosis (PGD) is a complex, multiphase process for couples, a new study has found. Understanding it, the investigators say, may be helpful to
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/AAN-logo-w-petriedishes.JPG){kind=logo}
May 09, 2012
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.
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/Lightswitch_old.JPG)
May 06, 2012
Researchers in Italy and Japan, supported in part by MDA, have identified what they believe is a molecular "switch" that may be inappropriately activating several genes in facioscapulohumeral
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/research_word_in_dictionary_magnified_1.jpg)
May 04, 2012
Charcot-Marie-Tooth disease (CMT) is a peripheral nerve disorder that can be caused by mutations in more than 50 different genes. Recent research has resulted in several new tools that will help
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/blood-vial-and-test-tubes.jpg)
May 02, 2012
The Muscular Dystrophy Association has awarded $1,549,725 to ReveraGen BioPharma, a Rockville, Md., biotechnology company, for development of a dissociative glucocorticoid to treat Duchenne muscular
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/AAN-logo-w-beakers.JPG){kind=logo}
May 02, 2012
The 2012 annual meeting of the American Academy of Neurology, held in New Orleans April 21-28, included findings related to myasthenia gravis (MG).
Eculizumab safe and effective in severe MG
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/Podcast_Mendell_Jerry.JPG)
May 01, 2012
An April 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores the implications of a recently developed strategy for newborn screening for Duchenne muscular dystrophy (DMD).
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/AAN-logo-w-beakers.JPG){kind=logo}
April 27, 2012
Among the reports given at the 2012 annual meeting of the American Academy of Neurology, being held April 21-28 in New Orleans, was information about research into amyotrophic lateral sclerosis (ALS
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/AAN-logo-w-microscope.jpg){kind=logo}
April 26, 2012
Update (June 1, 2012)— The brief Drugs help DMD-related cardiomyopathy has been updated to include a May 2012 podcast provided by Nationwide Children's Hospital's "This Month in Muscular Dystrophy
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/CheckmarkGreen_RedBox.JPG)
April 24, 2012
Update (June 21, 2012):This story was updated to include the fact that MDA funding helped support the phase 1 trial, as well as the preclinical development of RG3039.
RG3039, an experimental
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/test-tube-capsule-small.jpg)
April 23, 2012
Update (May 25, 2012) —Summit announced today that the first group of healthy volunteers in the phase 1 trial of its newly formulated SMT C1100 has begun receiving the drug. The dose-escalating trial
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/AAN%20logo%20w%20petriedishes_0.JPG)
April 23, 2012
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
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/test-tubes-illustration_0.jpg)
April 12, 2012
SOD1 causes toxicity in some familial and some sporadic ALS
In some people with bulbar-onset sporadic ALS, changes to the SOD1 protein cause the same toxicity as is generated by mutated SOD1 protein
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/2mice_0.jpg)
April 12, 2012
New research suggests that a potential strategy for treating Duchenne muscular dystrophy (DMD) may be to increase levels of heat shock proteins inside muscle fibers.
Heat shock proteins exist
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/6%20New%20Grants_0.jpg)
April 09, 2012
Preventing, halting or reversing motor neuron damage is a primary goal of ALS research. But new ways of thinking about the disease are guiding scientists down different roads to that destination.
MDA
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/ClinicalConf2012_ValerieCwik.JPG)
April 05, 2012
The progress of several experimental therapies currently in development for neuromuscular diseases was discussed at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.
Most of these emerging
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/Human_silhouette.JPG)
April 03, 2012
Neuralstem's spinal cord stem cells, and the surgical technique used to transplant them, proved to be safe and well-tolerated in a phase 1 clinical trial in 12 people with amyotrophic lateral
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/mouse-pill-bottle.JPG)
April 03, 2012
The Muscular Dystrophy Association has awarded $120,000 to Cambridge, Mass.-based Catabasis Pharmaceuticals as part of a strategic partnership under which the pharmaceuticals company will test two
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/heart_and_heartbeat_0.jpg)
April 02, 2012
New evidence suggests that relatively aggressive management of seemingly minor cardiac conduction defects in adults with type 1 myotonic dystrophy (MMD1, or DM1) can prolong survival.
Slowing of
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/researcher_writing%20copy.JPG)
April 02, 2012
Editor's note: This story was updated on April 27, 2012, and revised April 3, 2012.
Weekly intravenous administrations of the experimental exon-skipping agenteteplirsen (AVI-4658) in a U.S. trial
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/IVdrip_rubberglove.jpg)
March 29, 2012
The American Academy of Neurology (AAN) has released new guidelines on the use of a treatment called intravenous immunoglobulins (IVIG) in various neuromuscular disorders.
The disorders in MDA's
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/DNA_helix_multicolor.JPG)
March 27, 2012
Adequate levels of zinc finger protein 1 (ZPR1) appear to be a "protective" modifier of spinal muscular atrophy (SMA), an MDA-supported team of scientists has reported.
Modifiers influence disease
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/computer%20keyboard.JPG)
March 23, 2012
An overview of the development of TDI 132 for use in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) was the topic of a webinar hosted March 12, 2012, by the nonprofit biotech ALS
Pages
Connect with MDA
Muscular Dystrophy Association — USA
National Headquarters
3300 E. Sunrise Drive
Tucson, AZ 85718
(800) 572-1717
©2013, Muscular Dystrophy Association Inc. All rights reserved.
