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Last updated on April 19, 2014 at 9:20 EDT

Latest Survival of motor neuron Stories

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...

2014-01-22 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...

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...

2013-11-12 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...

2013-10-03 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...