Research News
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/Doc%27sHandsWritingLeadArt.jpg)
September 15, 2011
Update (July 25, 2012): This story was updated to reflect the fact that PTC has announced its intention to begin an open-label trial of ataluren in DMD/BMD for former ataluren trial participants in
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/globe-man-laptop-info-arc%20copy.jpg)
September 09, 2011
FUS and TDP43 work together in fruit flies
In a fruit fly research model of inherited ALS, human FUS and TDP43 proteins work together on some processes necessary for the long-term survival of muscle-
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/research_leadart.jpg)
September 01, 2011
Disease 'in a dish' is a new tool for studying ALS
An MDA-supported research team has created a human cellular model of ALS using cells taken from people with the ALS8 subtype of the disease, a
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/stoppingals_leadart.jpg)
September 01, 2011
In its latest round of funding this July, MDA awarded nine grants totaling more than $2.5 million to research projects focused on uncovering the causes of, and developing therapies for, amyotrophic
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/AstrocytesLeadArt.JPG)
August 29, 2011
Astrocytes— cells that normally support and protect nerve cells — have been found to cause motor neuron degeneration in newly created human cellular models of both inherited and noninherited ALS (
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/DNAmodel_redspot.jpg)
August 25, 2011
A mutation in the gene for a protein called sigma intracellular receptor 1 (sigma R1) has been identified as a cause of familial juvenile ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/DNA%20strand%2CLeadArt.jpg)
August 22, 2011
A group of researchers led by Teepu Siddique at Northwestern University Feinberg School of Medicine in Chicago has identified abnormalities in the ubiquilin 2 gene and protein as important
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/GrantsDollars.jpg)
August 22, 2011
MDA has awarded nine grants totaling more than $2.5 million to fund research projects focused on uncovering the causes of, and developing therapies for, amyotrophic lateral sclerosis (ALS, or Lou
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/test_tube_dollar_0.jpg)
August 22, 2011
The Muscular Dystrophy Association has awarded 40 research grants totaling $13.7 million to advance the understanding of disease processes and uncover new strategies for treatments and cures of
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/FKRPregistry_WorldwideNetwork.jpg)
August 19, 2011
A new registry has been launched for people with conditions caused by mutations in the gene for fukutin-related protein (FKRP). This includes people with the type 2I form of limb-girdle muscular
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/test-tubes-with-blue-caps_0.jpg)
August 15, 2011
Updated: View MDA's video about the launch of the eteplirsen trial on YouTube.
Biotechnology company AVI BioPharma announced Monday, Aug. 15, 2011, that its phase 2 exon-skipping trial of eteplirsen
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/free%20weight.jpg)
August 08, 2011
The National Institute of Neurological Disorders and Stroke (NINDS), part of the U.S. National Institutes of Health (NIH), is conducting a study to assess the safety and effectiveness of exercise in
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/2mice.jpg)
August 08, 2011
The administration of a protein called prolactin has been shown to slow weight loss, improve motor function and increase life span by approximately 70 percent in mice with a disease resembling a
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/groups%20gathering.jpg)
August 05, 2011
The 2011 Periodic Paralysis Association Conference will be held Nov. 4-6 in Orlando, Fla., and is geared toward people with periodic paralysis (PP), their families, physicians and friends.
This is
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/QNO%20windows%20open.jpg)
August 04, 2011
The "window of opportunity" for treating infants at risk of developing spinal muscular atrophy (SMA) may not be as narrow as some experts have feared, new experiments in mice suggest.
MDA research
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/mouse-testing-LeadArt.jpg)
July 28, 2011
An experimental compound called trichostatin A (TSA) has been shown to improve nerve-cell survival, increase strength and muscle weight, and result in longer life span in mice with a disease
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/DoubleHelix%20and%20scope.jpg)
July 26, 2011
Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD)
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/syringes%2C%20trio.jpg)
July 22, 2011
Biotechnology company BrainStorm Cell Therapeutics has announced plans to collaborate with two American institutions to test its experimental stem cell technology in people with amyotrophic lateral
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/DMD%2CPrednisoneLeadArt.jpg)
July 20, 2011
A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/ALS%20registry%20LeadArt.jpg)
July 14, 2011
In hopes of collecting as much information as possible, the National ALS Registry, which opened in October 2010, already is expanding its reach.
Registry officials have made the enrollment process
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/man-on-globe-with-Laptop%20copy_0.jpg)
July 07, 2011
Edison drugs target FA, mitochondrial diseases
Edison Pharmaceuticals, a biotechnology company, announced in June 2011 that its experimental drug EPI-A0001 has shown promise in a 28-day, placebo-
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/5strategies_leadart.jpg)
July 01, 2011
Antisense oligonucleotides block flawed genetic instructions
Antisense oligonucleotides — also called antisense, oligos, or simply AONs— are pieces of genetic code that keep other genetic code from
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/cmt_today_leadart.jpg)
July 01, 2011
MDA-supported research in Charcot-Marie-Tooth disease is focused on figuring out what goes wrong at the molecular level in CMT-affected axons or the myelin sheaths that surround them, rather than on
![[title]](http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/world_network.jpg)
July 01, 2011
The field of Charcot-Marie-Tooth disease (CMT) research is expanding, and people with the disease can help move it forward.
Discovery of the complicated genetics underlying CMT has made it clear that
![[title]](http://alsn.mda.org/files/alsn/imagecache/175xFrontFeature/scientists_like_leadart.jpg)
July 01, 2011
Karen Felzer studies earthquakes, not ALS.
But after her father learned he had the disease, Felzer helped conduct a study that caused a small shake-up in the world of ALS research.
Working with
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.
