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Latest SMN1 Stories

2014-08-15 08:24:22

CARLSBAD, Calif., Aug. 15, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $2 million milestone payment from Biogen Idec related to the advancement of the ongoing open-label extension study of ISIS-SMN(Rx) in children with spinal muscular atrophy (SMA). http://photos.prnewswire.com/prnvar/20130807/LA60006LOGO The open-label extension study of ISIS-SMN(Rx) is offered to those children with SMA who have completed dosing in Isis'...

2014-08-07 16:27:47

-Results published in Science show small molecule SMN2 splicing modifiers to be effective in SMA mouse models- SOUTH PLAINFIELD, N.J., Aug. 7, 2014 /PRNewswire/ -- Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth is preventing deficits in mouse models of Spinal Muscular Atrophy (SMA). Scientists from Roche Pharma Research and Early Development...

2014-08-01 08:25:54

-- ENDEAR Trial To Enroll 110 SMA Infants at Sites Globally CARLSBAD, Calif., Aug. 1, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMN(Rx) in infants with spinal muscular atrophy (SMA), the most common genetic cause of infant mortality. Isis plans to dose the first infant in this study within the next few weeks, at which time Isis will earn an $18 million milestone payment from its...

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...

2014-02-11 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 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...

2013-12-09 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...


Word of the Day
bodacious
  • Remarkable; prodigious.
  • Audacious; gutsy.
  • Completely; extremely.
  • Audaciously; boldly.
  • Impressively great in size; enormous; extraordinary.
This word is probably from the dialectal 'boldacious,' a blend of 'bold' and 'audacious.'
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