Quest Magazine

AAN Research Briefs on DM, DMD, LGMD, MG, MMD, SMA

Below are brief reports and links to more information about neuromuscular disease research presented at the 63rd annual meeting of the American Academy of Neurology (AAN), held in Honolulu April 9-16, 2011.

ALS Research Briefs from 2011 AAN Meeting

Neuralstem's trial of spinal cord injections of stem cells is on track

MDA Surveying Families about ‘Cost of Illness’

Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak to policy makers.

MDA Surveying Families about ‘Cost of Illness’

Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak to policy makers.

Scholarships, Grants Available to People with Disabilities

(Update 9/14/11: The 2011 scholarship winners have been selected. To view a list of winners visit www.deshae.org/cmms/awards/2011/scholars.pdf. Applications for quality of life grants continue to be accepted and are awarded on an ongoing basis.)

Free eye exams for service dogs

The American College of Veterinary Ophthalmologists (ACVO) is hosting the 4thAnnual ACVO/Merial National Service Dog Eye Exam Event this May.

Thousands of service dogs will receive free eye examinations at participating veterinary ophthalmology clinics throughout the United States, Canada and Puerto Rico. Last May, more than 3,200 service dogs participated in the event. Registration is now open; appointments are given on a first-come, first-served basis.

Phase 3 Trial of Dexpramipexole Launched in ALS

Update 8/31/11: Enrollment screening for this study has closed to new prospective participants.

Research Briefs: DMD Exon Skipping

sClinical trials that use compounds called antisense oligonucleotides to cause skipping of exon 51 of the dystrophin gene in individuals with Duchenne muscular dystrophy (DMD) are moving forward in the United States and elsewhere.

Exon skipping for DMD is a strategy that coaxes muscle fibers to ignore, or "skip," the genetic instructions for certain parts of the dystrophin gene so that functional dystrophin protein can be made despite the presence of a genetic mutation.

MDA Awards Grant to Glialogix to Test Potential ALS Treatment

MDA is funding research into a potential treatment for amyotrophic lateral sclerosis (ALS) that inhibits excessive glutamate signaling in the brain and spinal cord.

DMD/BMD Drug Screen in Zebrafish Finds Three Candidates

Three small molecules that normalize muscle structure and function and improve survival in dystrophin-deficient zebrafish have been identified by scientists at Children's Hospital Boston, Harvard Medical School, and Alaska Fisheries Science Center in Juneau.

The three chemicals, which already are approved for other medical uses, were found to increase survival and normalize muscle structure and function in the dystrophin-deficient fish without increasing  production of the muscle protein dystrophin. 

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