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Last updated on April 21, 2014 at 9:29 EDT

Latest Spinal muscular atrophy Stories

2013-12-18 15:08:14

Research team identified Neurexin2 as a novel target for potential therapy of neurodegeneration in Spinal Muscular Atrophy patients A recent study led by scientists from the National University of Singapore (NUS) opens a possible new route for treatment of Spinal Muscular Atrophy (SMA), a devastating disease that is the most common genetic cause of infant death and also affects young adults. As there is currently no known cure for SMA, the new discovery gives a strong boost to the fight...

2013-12-09 23:22:43

It describes the scientific strategy behind Antisense Drugs. It also provides updates on the ongoing research and clinical trials for this class of SMA drugs. Elk Grove Village, IL (PRWEB) December 09, 2013 Families of SMA has released a new edition of the Compass Newsletter, a publication dedicated to research updates. This newsletter was produced in a collaboration between Families of SMA, Biogen Idec, and Isis Pharmaceuticals. It describes the scientific strategy behind Antisense...

2013-11-22 08:23:05

CARLSBAD, Calif., Nov. 22, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it plans to add a 12 mg cohort to the ongoing Phase 1b/2a study evaluating ISIS-SMN(Rx) in children with spinal muscular atrophy (SMA). In addition, a 12 mg dose will be included in an open-label extension study for those children who have completed dosing in previous studies. The addition of the 12 mg cohort will allow for the investigation of this dose in support of the...

2013-11-18 23:24:29

FightSMA Thrilled to Make Announcement After Years of Advocacy by Families Across the U.S. Washington, DC (PRWEB) November 18, 2013 FightSMA (http://www.fightsma.org) is tremendously proud to announce that after years of passionate advocacy by families across the country the United States Senate passed the National Pediatric Research Network Act (NPRNA) Nov. 14 by unanimous consent.  The legislation, which the U.S. House approved on Tuesday, Nov.12, now heads to the President's...

2013-11-14 08:33:24

-- Conference call to be held today at 8:00 a.m. ET -- SOUTH PLAINFIELD, N.J., Nov. 14, 2013 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes, today announced financial and corporate results for the quarter ended September 30, 2013. (Logo: http://photos.prnewswire.com/prnh/20010919/PTCLOGO ) "2013 has thus far...

2013-11-13 23:01:06

Bill authorizes the National Institutes of Health (NIH) to support the creation of up to 20 pediatric research consortia focused on diseases such as Spinal Muscular Atrophy. Washington, DC (PRWEB) November 13, 2013 National legislation to advance spinal muscular atrophy research took a giant leap forward on Tuesday, Nov. 12, 2013 with the passage by the U.S. House of Representatives of the National Pediatric Research Network Act (NPRNA). The NPRNA has been supported by a coalition of...

2013-11-12 08:34:13

CARLSBAD, Calif., Nov. 12, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $1.5 million milestone payment from Biogen Idec related to the advancement of the ongoing Phase 2 study of ISIS-SMN(Rx) in infants with spinal muscular atrophy (SMA). (Logo: http://photos.prnewswire.com/prnh/20130807/LA60006LOGO) The Phase 2 study of ISIS-SMN(Rx) is an open-label, multiple-dose, dose-escalation pilot study in infants who have been diagnosed...

2013-10-31 23:37:18

Stanton Smiles“Dr. Robert B Stanton DMD” and his friends are attending The Gala of Hope. A South Florida fundraising event with one goal: To fund a cure for Spinal Muscular Atrophy. Fort Lauderdale, FL (PRWEB) October 31, 2013 In his never-ending pursuit to help his community, Dr. Robert B Stanton is joining the fight against Spinal Muscular Atrophy (SMA) by donating a Zoom Whitening Treatment, buying tickets to the Gala and participating in the auction. The money raised from his...

2013-10-16 08:26:49

CARLSBAD, Calif., Oct. 16, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $10 million milestone payment from Biogen Idec related to the selection and advancement of ISIS-DMPK(Rx) to treat myotonic dystrophy type I (DM1). (Logo: http://photos.prnewswire.com/prnh/20130807/LA60006LOGO) "We are very pleased with the successes we are having in our alliance with Biogen Idec. The progress we have made is evidenced in our spinal...

2013-10-03 16:23:10

--Isis earns $2 million milestone payment-- CARLSBAD, Calif., Oct. 3, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it dosed the first infant in the 12 mg dose group in the Phase 2 study evaluating ISIS-SMN(Rx) in infants with Type I spinal muscular atrophy (SMA). The Phase 2 study is designed to allow Isis to define the optimal dose for the larger Phase 2/3 study in infants and to provide safety and tolerability data. Isis plans to complete...