Sentences with phrase «cure sma»
«We are encouraged by the preclinical data emerging on SRK - 015, including the effects upon fast - twitch muscle fibers that are particularly relevant for SMA as well as its selectivity profile, which may be very important when considering chronic therapy in children,» said Karen S. Chen, PhD, Chief Scientific Officer of the SMA Foundation and a co-author of the study being presented at the Cure SMA Annual Conference.
http://www.smafoundation.org/
As director of the pediatric motor disorders research program at the University of Utah School of Medicine in Salt Lake City, she led the Project Cure SMA Investigators Network, a trial consortium that recently published the results of two placebo - controlled trials of the HDAC - blocking agent valproic acid, neither of which demonstrated much clinical advantage from the drug1, 2.
The next step will be to try to cure the SMA in the dish by replacing defective genes or screening for an effective drug.
Sign the petition for Funding of Spinraza for all Canadian patients affected by Spinal Muscular Atrophy (SMA) Cure SMA Canada, CORD, the Canadian SMA families and our supporters appeal to our provincial health ministers to provide all patients access to the only treatment available for this life threatening disease.
Which is why Cure SMA Canada and the Canadian Organization for Rare Disorders are calling upon all Canadians to sign this petition, demanding governments include all SMA patients in the funding of Spinraza and to make the drug available immediately.
Not exact matches
Although there is no cure for SMA currently, the National Institutes of Health (NIH) has listed SMA as the neurological disease closest to finding a cure, due in part to effective drugs like the one developed in Lorson's lab.
Mostly caused by an inherited flaw in the Survival Motor Neuron (SMN) gene, SMA is presently without a cure.
New York, NY — November 29, 2004 — The Spinal Muscular Atrophy Foundation, a nonprofit organization dedicated to finding a treatment or cure for spinal muscular atrophy (SMA)-- the leading genetic killer of infants and toddlers — announced that is has funded more than $ 15 million in research in the last 18 months.
The SMA Foundation has diverse drug discovery collaborations with both academia and the private sector to accelerate the development of a treatment or cure.
Through regional networks in the U.S. like the Pediatric Neuromuscular Clinical Research (PNCR) Network and Project Cure, and international organizations such as the International Coordinating Committee (ICC) and TREAT - NMD, the SMA community has collaborated effectively on recruiting patients, establishing standards of care, standardizing outcome measures, and supporting multi-site clinical efforts, such as the Biomarkers for SMA (BforSMA) and the PNCR Network Natural History studies.
The SMA gene replacement therapy developed by the Mendell and Kaspar teams at Nationwide Children's Hospital and collaborators at Ohio State University has the realistic potential to cure a devastating neurological disorder of newborn infants.
About SMA Foundation The SMA Foundation is a nonprofit organization, founded in 2003, dedicated to finding a treatment and potential cure for Spinal Muscular Atrophy (SMA).
The research network augments the Foundation's aggressive agenda to fund research initiatives which will accelerate the development of a treatment or cure for SMA.
Founded in 2003, the SMA Foundation is a nonprofit organization dedicated to accelerating progress towards a treatment and cure for Spinal Muscular Atrophy (SMA) through targeted funding of clinical research and novel drug development efforts.
There is no cure and not a single proven treatment for SMA.
The SMA Foundation is a nonprofit organization founded in 2003 dedicated to finding a treatment or potential cure for spinal muscular atrophy (SMA).
In February, we selected the charity which will benefit from all profit on the event: The SMA Trust, a children's charity which supports vital research into finding a cure for Spinal Muscular Atrophy.
The conference raised over # 10,000 for the SMA Trust, a children's charity which supports vital research into finding a cure for Spinal Muscular Atrophy.