Research News
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/computer_survey.jpg)
April 02, 2013
An online survey about ALS symptoms, now open to people with amyotrophic lateral sclerosis (ALS), is being conducted by the Neurological Clinical Research Institute at Massachusetts General Hospital
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/neurons.jpg)
March 29, 2013
Scientists continue to work at uncovering the biological mechanisms underlying amyotrophic lateral sclerosis (ALS). One area of intense study involves the potential role of central nervous system
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_100x75/computer_screen_with_magnifier_3.jpg)
March 26, 2013
Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.
DMD: Building muscle, fighting inflammation
DART
![[title]](http://alsn.mda.org/files/alsn/imagecache/mda_org_100x75/research_alsn.jpg)
March 22, 2013
The latest advances in brain and central nervous system research were discussed at the 65th annual meeting of the American Academy of Neurology, held in San Diego, March 16-23, 2013.
Reports included
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/test-tubes_hands.JPG)
March 21, 2013
An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.
The drug, ISIS-SMNRx, is being developed and
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/StemcellLeadArt_0.jpg)
March 20, 2013
A therapeutic strategy that combines gene therapy and stem cell transplantation has shown encouraging results in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).
"Our findings
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/fatherand%20newborn_0.jpg)
March 20, 2013
Proposals exploring the feasibility and advisability of implementating newborn screening for two disorders in MDA's program — Duchenne muscular dystrophy and Pompe disease— were presented to a
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/brain_from_above%20alsn.jpg)
March 15, 2013
Advances in technology have led to greater sensitivity in imaging techniques, increasing scientists' ability to see into the brain and spinal cord. Now, a research team reports that a technique
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/microscope_closeup2_1.jpg)
March 14, 2013
Spinal muscular atrophy (SMA) is a disease in which nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles. Recent research
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/DoubleHelix_WorldMap_1.JPG)
March 12, 2013
An international patientregistry (database) is gathering information about children and adults with any form of congenital muscular dystrophy (CMD), a congenital myasthenic syndrome or a congenital
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/people-cutouts.jpg)
March 08, 2013
The number of new cases of amyotrophic lateral sclerosis (ALS) identified each year (incidence) and the number of people living with the disease (prevalence) appears to be lower in American Indians
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/2010laspada01a.jpg)
March 03, 2013
Mice with a disorder mimicking human spinal-bulbar muscular atrophy(SBMA, or Kennedy disease) that were treated with an experimental therapy called arimoclomol showed improved nerve-cell survival,
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/fa_cantera-leadartV1.jpg)
February 28, 2013
Idebenone (under the brand name Catena) in July 2008 received conditional market approval in Canada for the treatment of Friedreich's ataxia (FA). Now, based on additional data that fails to confirm
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/molecule-model-being-built.jpg)
February 27, 2013
Scientists at the biopharmaceutical company Genzyme, working with mice, say they have modified and improved an existing experimental strategy to treat type 1 myotonic muscular dystrophy (MMD1, also
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/ptc_peltz_LeadArt.jpg)
February 21, 2013
Trials of the experimental muscular dystrophy drug ataluren showed that the drug was generally well-tolerated and slowed the rate of decline in walking ability, compared to the placebo group.
After
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/test-tubes-capsule-illustration_0.jpg)
February 15, 2013
The experimental drug AT2220 has shown benefit as an enhancer of enzyme replacement therapy for the metabolic muscle disorder Pompe disease (acid maltase deficiency).
The drug, a pharmacological
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/blue-pill-case.jpg)
February 15, 2013
Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/newborn_baby.jpg)
February 13, 2013
Update (March 28, 2013): This story was updated with a link to the ClinicalTrials.gov site for this study.
Researchers conducting a study of natural history and biomarkers in infants with type 1
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/dna_blue_helix_tech.jpg)
February 11, 2013
MDA has awarded a $400,000 grant to National Institutes of Health (NIH) Laboratory of Neurogenetics researchers to perform exome sequencing on samples taken from 1,000 people with sporadic
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/test-tube-colors-semicircle.jpg)
February 10, 2013
The U.S. Food and Drug Administration (FDA) has given the go-ahead to the nonprofit biotech ALS Therapy Development Institute (ALS TDI) to conduct a clinical trial of TDI132 — also known as
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/blue-capsulesLeadArt.jpg)
February 06, 2013
A 12-participant study of the acute effects of two vasodilating drugs on blood flow to exercising muscles needs four more boys with Duchenne muscular dystrophy (DMD) who meet study criteria and are
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/gold_dollars.jpg)
February 04, 2013
Twelve new grants totaling $3.6 million have been awarded in support of research studies that will explore the causes of, and potential treatments for, amyotrophic lateral sclerosis (ALS).
“This is
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/piechart%2Cpeople.jpg)
February 03, 2013
A recently opened global registry— a database of patient information — is seeking people with the type 2A form of limb-girdle muscular dystrophy (LGMD2A), a form of LGMD that results from a
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/microscope_closeup_alsn.jpg)
January 25, 2013
Development of stem cell therapy to treat amyotrophic lateral sclerosis (ALS) continued with two studies in mice, one by an Israeli biotech company and one by an Italian research team.
Repeat doses
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/http%20survey.jpg)
January 23, 2013
A survey of people with inclusion-body myositis (IBM) is being conducted by A. David Paltiel, a professor of public health (health policy) and management at Yale University, with colleagues there and
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.
