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Latest Spinal muscular atrophy Stories

2014-04-11 23:03:45

Each winner receives $27,500 for Spinal Muscular Atrophy (SMA) research. Washington, DC (PRWEB) April 11, 2014 Two organizations dedicated to defeating the childhood neuromuscular disorder spinal muscular atrophy (SMA) announced today three winners of the “FightSMA and Gwendolyn Strong Foundation Emerging Investigator Awards.” The awards, conceived jointly by the two organizations, are designed to draw strong, innovative talent into SMA research, according to FightSMA Chair Mike...

2014-03-06 16:27:15

-Conference Call Today at 4:30 pm ET- SOUTH PLAINFIELD, N.J., March 6, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported its financial results for the fourth quarter and full-year ended December 31, 2013. http://photos.prnewswire.com/prnvar/20010919/PTCLOGO "2013 was a landmark year for PTC with significant accomplishments achieved across the organization. Our private financing early in the year combined with a very successful IPO...

2014-02-21 23:04:33

These funds were awarded as part of Families of SMA’s basic research grant program. Basic research looks into the causes of SMA, revealing new and better ways of making drugs to target the disease. Elk Grove Village, IL (PRWEB) February 21, 2014 Families of SMA has announced seven grants, totaling $600,000 in new funding, for research into the causes of Spinal Muscular Atrophy (SMA). SMA affects an estimated 1 in every 10,000 children, and is the number one genetic killer of children...

2014-02-21 16:23:09

On track to initiate Phase 3 study in infants with SMA in the middle of this year CARLSBAD, Calif., Feb. 21, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today provided an update on its ongoing open label, multiple dose Phase 2 study of ISIS-SMN(Rx) in infants with spinal muscular atrophy (SMA). To date, all four infants in the 6 mg cohort have been in the study for over six months and are now approximately nine and a half to 16 months in age with an average age...

2014-02-21 08:25:03

Average increase of 3.7 points observed in muscle function score in SMA children treated with 9 mg of ISIS-SMN Rx CARLSBAD, Calif., Feb. 21, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced top-line results from an ongoing open-label, multiple-dose study of ISIS-SMN(Rx )in children with spinal muscular atrophy (SMA). In this study, ISIS-SMN(Rx) was well tolerated at all dose levels in children with SMA. Consistent with single-dose observations,...

2014-02-13 08:30:17

--Isis will receive $9.3 million in milestone payments -- CARLSBAD, Calif., Feb. 13, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that the first child in the 12 mg group was dosed in the ongoing Phase 2 study evaluating ISIS-SMN(Rx) in children with spinal muscular atrophy (SMA). Isis also announced today that the first child was dosed in an open-label extension study, which is being offered to those children with SMA who have completed dosing in...

2014-02-11 23:20:57

Use of Genetic Variants Improves Detection of SMA Carriers in Clinical Test that will be Broadly Licensed to Testing Labs Worldwide New York, NY (PRWEB) February 11, 2014 The Icahn School of Medicine at Mount Sinai today announced the launch of a more accurate carrier screening test for spinal muscular atrophy (SMA), one of the most common and severe autosomal recessive disorders. This new test will help prospective parents more effectively identify whether they carry the mutation that...

2014-01-22 23:04:23

Program Created by FightSMA and Gwendolyn Strong Foundation to Advance Spinal Muscular Atrophy Research Washington, DC (PRWEB) January 22, 2014 The "FightSMA and Gwendolyn Strong Foundation Emerging Investigator Awards" program (EIA Program) announced this week a second year of grant submissions and selection. The awards are funded by a grant provided by the two organizations created to attract a new generation of investigators into the spinal muscular atrophy (SMA) research...

2014-01-22 00:21:53

SOUTH PLAINFIELD, N.J., Jan. 22, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), the SMA Foundation, and Roche (SIX: RO, ROG; OTCQX: RHHBY), announced today that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the...

2014-01-15 23:04:39

The organizations conceived the awards in order to draw strong, innovative talent into Spinal Muscular Atrophy research. Alexandria, VA (PRWEB) January 15, 2014 Two organizations, allied in the fight to defeat the deadly childhood disease spinal muscular atrophy (SMA), jointly announced this week the winners of the “FightSMA/Gwendolyn Strong Foundation Emerging Investigator Awards.” FightSMA and the Gwendolyn Strong Foundation conceived the awards in order to draw strong, innovative...


Word of the Day
grass-comber
  • A landsman who is making his first voyage at sea; a novice who enters naval service from rural life.
According to the OED, a grass-comber is also 'a sailor's term for one who has been a farm-labourer.'