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
December 16, 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
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
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
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
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
December 05, 2012
Update (Dec. 6, 2012): This story has been updated with additional information about next steps for ataluren.
New Jersey biopharmaceutical company PTC Therapeutics has announced that the European
December 04, 2012
Protein test predicts ALS progression
It’s known that levels of a protein called phosphorylated neurofilament heavy subunit (PNF-H) are increased in the spinal fluid of people with amyotrophic
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,
November 28, 2012
The drug tadalafil (Cialis, Adcirca), which dilates blood vessels and is approved to treat erectile dysfunction and pulmonary hypertension, has been found to improve blood flow to exercising forearm
November 26, 2012
Update (Dec. 20, 2012):This story has been updated to reflect the availability of a 12-minute podcast with researcher Michael Rudnicki, in which Rudnicki discusses the advantages and disadvantages of
November 25, 2012
Immune system cells known as regulatory T cells— T-regs for short — have been found to play a protective role in mice with a disorder that resembles human amyotrophic lateral sclerosis (ALS). They
November 19, 2012
Update (Dec. 4, 2012):This story was updated to reflect that the 13 previously identified genes that were tested as potentially associated with Adam Foye's disease were genes for various disorders
November 18, 2012
Findings from a study of 516 Italian boys show a significant improvement over the last six decades in survival time in Duchenne muscular dystrophy (DMD). This modified natural history (general
November 15, 2012
Update (Feb. 13, 2013):In February 2013, study coordinator Lynne Wolfe at the National Institutes of Health said the NIH study of EPI-743 in metabolic and mitochondrial disordersis seeking children
November 15, 2012
A phase 1b/2a trial to test the safety and tolerability of multiple doses of the experimental drug ISIS-SMNRx in children with spinal muscular atrophy (SMA) is now open at sites in New York and Salt
November 13, 2012
Nerve cells called serotonergic neurons degenerate in amyotrophic lateral sclerosis (ALS) and may influence the ALS disease process, an international research team has reported. In particular, the
November 11, 2012
A webinar (Web-based seminar) about neurodegenerative causes of death, including amyotrophic lateral sclerosis (ALS), in retired National Football League (NFL) football players is scheduled for 1 p.m
November 11, 2012
Update (Nov. 14, 2012): This story has been updated to reflect information about genetic testing.
Scientists funded in part by MDA have found the cause of type 2facioscapulohumeral muscular
November 04, 2012
Results from a completed phase 2 trial of the experimental therapy NP001 in people with amyotrophic lateral sclerosis (ALS) show that in some trial participants, the drug appeared to be effective at
November 02, 2012
The multinational pharmaceutical company GlaxoSmithKline (GSK) has announced promising results for its phase 1 trial of the exon-skipping drug drisapersen in boys with Duchenne muscular dystrophy (
October 28, 2012
Update (Feb. 1, 2013): Dutch biopharmaceutical company Prosensa announced Jan. 29 that it has received orphan drug status in the United States and the European Union for compounds in development for
October 25, 2012
It's been known since the early 1990s that mutations in the superoxide dismutase-1 (SOD1) gene can result in the production of any number of varieties of improperly folded (misfolded) SOD1 protein,
October 25, 2012
Investigators at Northwestern University in Chicago are seeking people with spinal muscular atrophy (SMA), ages 2 to 21, to participate in a study about preferences and involvement in recreational
October 25, 2012
Researchers funded in part by MDA say a gene-based therapy designed to treat myasthenia gravis (MG) has shown promise in mice with an MG-like disease.
The research team was led by Dan Drachman, a