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Research News

[title]
Study of Tadalafil in BMD Revised, Reopened
October 02, 2012
Update (Nov. 9, 2012): This story has been updated to reflect that recruitment for this study ends Dec. 31, 2012. An MDA-supported, short-term trial of the vasodilating drug tadalafil (Cialis) in
[title]
SMA: Repligen Launches Phase 1b Trial of RG3039
October 02, 2012
The experimental drug RG3039, being developed by Repligen Corp. of Waltham, Mass., as a potential treatment for spinal muscular atrophy (SMA), has moved into its next phase of testing. RG3039
[title]
ALS-FTD Research Briefs: P62 Gene’s Role and Screening Tool for ALS-FTD
September 25, 2012
The most visible symptom in amyotrophic lateral sclerosis (ALS) is progressive weakness and loss of muscle control due to the loss of nerve cells called motor neurons. But approximately half of all
[title]
Study Recruiting People with LGMD2B or Miyoshi Myopathy
September 25, 2012
A new, multinational study of type 2B limb-girdle muscular dystrophy (LGMD2B) and Miyoshi myopathy— both of which result from mutations in the gene for the muscle protein dysferlin and are known as
[title]
New Phase 2b Trial of Tirasemtiv (CK-2017357) in ALS
September 19, 2012
Update (Oct. 29, 2012): This article has been updated to reflect the fact that the trial is now open to recruitment. South San Francisco biotechnology company Cytokinetics announced Sept. 10, 2012,
[title]
MDA Muscle Symposium: Newborn Screening in DMD
September 19, 2012
Update (Feb. 8, 2013): Longtime MDA grantee Jerry Mendell, who directs the Center for Gene therapy at Nationwide Children's Hospital in Columbus, Ohio, published Report of MDA Muscle Disease
[title]
NINDS Launches Clinical Trial Network, SMA Biomarkers Study
September 18, 2012
Update (March 28, 2013): This story was updated with a link to the ClinicalTrials.gov site for this study. The National Institute of Neurological Disorders and Stroke (NINDS) has launched a study to
[title]
DMD Briefs: Stopping Inflammation on Earth and in Space
September 13, 2012
Inflammation— the immune system's first line of defense in tissue that's been damaged by injury or infection — is a good example of a process that's a good thing up to a point and under certain
[title]
IGF1 Shows Benefit in SBMA Mice
September 09, 2012
Mice with a disease resembling spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) that were treated with a compound based on insulin-like growth factor 1 (IGF1) had better motor function,
[title]
BMD Conference Videos Cover Health Care, Research and Daily Living
September 07, 2012
Videos of the entire MDA-sponsored Becker Muscular Dystrophy Conference, held in Chicago on Aug. 11, 2012, are now archived on the Conference video page. Each presentation covers a different aspect
[title]
ALS Risk Elevated in Some Retired NFL Players
September 05, 2012
Update (Nov. 6, 2012):This story was updated to reflect the availability of an abstract of the scientific paper (online and print) on which the story is based, as well as information about an
[title]
ALS and SMA: Less EPHA4 Means More Motor Neurons Survive
September 02, 2012
Decreased activity of the EPHA4 receptor, which is encoded by the EPHA4 gene, increases life span in people with amyotrophic lateral sclerosis (ALS), a team of scientists has reported. The receptor,
[title]
Sarepta Issues ‘Open Letter’ Explaining Development of Eteplirsen for DMD
August 31, 2012
In July 2012, Sarepta Therapeutics announced encouraging interim results for its phase 2b trial of eteplirsen, an experimental exon-skipping drug in development to treat Duchenne muscular dystrophy (
[title]
Chemical 'Signature' May Be Used to Track ALS
August 28, 2012
A "pro-inflammatory" chemical signature displayed by monocytes (a type of white blood cell) appears to signal the presence of amyotrophic lateral sclerosis (ALS) even before symptoms begin, a team of
[title]
MDA Commits $10.7 Million to Neuromuscular Disease Research
August 20, 2012
The Muscular Dystrophy Association has awarded 33 new grants totaling $10,684,481 to fund research projects focused on uncovering the causes of, and developing therapies for, neuromuscular disease.
[title]
ALS: New MDA Grants Focus on Multisystem Aspects of the Disease
August 19, 2012
Three new MDA grants totaling $891,156 have been awarded to research projects aimed at uncovering some of the many complex processes that underlie ALS. "Scientists are digging ever deeper into the
[title]
Mitochondrial Disease Registry Seeks Participants
August 15, 2012
If you or someone in your family has or is suspected of having a mitochondrial myopathy or other disorder of the mitochondria, the North American Mitochondrial Disease Consortium (NAMDC) would like
[title]
DMD Research Briefs: Exon Skipping Advances in Trials, Lab
August 15, 2012
Exon skipping is an experimental therapeutic strategy in which regions — exons— of a gene are targeted and blocked ("skipped") by laboratory-designed molecules. The goal is that the remaining genetic
[title]
MDA Funds Efficacy Trial of Diaphragm Pacing System in ALS
August 13, 2012
The Muscular Dystrophy Association has committed $750,000 to help support a phase 2 clinical trial assessing the ability of the NeuRx Diaphragm Pacing System (DPS) to improve respiratory function and
[title]
DUX4 Protein Not Unique to FSHD-Affected Muscles
August 09, 2012
Researchers supported in part by MDA have recently found evidence that production of the full-length version of a protein known as DUX4— previously associated exclusively with facioscapulohumeral
[title]
'Repeats' in Normal C9ORF72 Gene Don't Affect ALS, FTD
August 09, 2012
Humans carry two copies of the C9ORF72 gene, each containing a stretch of DNA in which a sequence of DNA building blocks coded GGGGCC is repeated a number of times. (Each six-letter sequence is
[title]
Phase 3 Trial of Ceftriaxone in ALS Stopped
August 08, 2012
On Aug. 8, 2012, the National Institute of Neurological Disorders and Stroke (NINDS) reported that it has stopped a phase 3 clinical trial of the antibiotic ceftriaxone in amyotrophic lateral
[title]
New Gene Therapy Approach for FA, Other Diseases
August 07, 2012
Human cells treated with engineered transcription activation-like effector (TALE) proteins produced two to three times more frataxin protein than did control cells, a team of researchers has reported
[title]
Doctors Surprised By Which Symptoms Matter Most to Adults with MMD1, FSHD
August 05, 2012
Traditionally, outcome measures— the observations investigators make in a clinical trial to decide whether one treatment is better than another or better than a placebo — are determined by factors in
[title]
Survey Examines Newborn Screening for Neuromuscular Disease
August 02, 2012
MDA is conducting a survey designed to assess the experiences of parents in the United States whose babies underwent newborn screening at the time of birth, with an eye toward the future possibility

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