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Latest Spinal muscular atrophy 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...

2014-08-04 08:28:12

- $40 million in milestone payments from partners drives improved quarterly financial performance CARLSBAD, Calif., Aug. 4, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today reported pro forma net operating income of $1.1 million and a pro forma net operating loss (NOL) of $21.5 million for the three and six months ended June 30, 2014, respectively, compared to an NOL of $5.3 million and $799,000 for the same periods in 2013. Isis' pro forma operating income in the second...

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...

2014-06-09 08:28:10

Isis Earns $14 Million Milestone Payment from Biogen Idec CARLSBAD, Calif., June 9, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has initiated a Phase 1 study for ISIS-DMPK(Rx). Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement. ISIS-DMPK(Rx) is designed to reduce the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells, including muscle cells, for the treatment of...

2014-05-06 08:35:15

-Conference Call Webcast Tuesday, May 6, 11:30 a.m. ET at www.isispharm.com CARLSBAD, Calif., May 6, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today reported a pro forma net operating loss (NOL) of $22.6 million for the three months ended March 31, 2014 compared to pro forma operating income of $4.5 million for the same period in 2013. Isis' first quarter 2014 pro forma NOL was higher than the same period last year primarily due to variations in the timing of milestone...

2014-04-30 08:32:02

CARLSBAD, Calif., April 30, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that the strategic alliance between Isis and Biogen Idec (NASDAQ:BIIB) has been honored with the Alliance 2014 Breakthrough Award. The award, sponsored by Thomson Reuters Recap, was presented at the Allicense 2014 Conference in San Francisco on April 29, 2014. http://photos.prnewswire.com/prnvar/20130807/LA60006LOGO "We are grateful for the award and recognition of our...

2014-04-29 12:34:49

Results presented at the American Academy of Neurology meeting CARLSBAD, Calif., April 29, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today provided an update on both of its ongoing open-label Phase 2 clinical studies of ISIS-SMN(Rx) in infants and children with spinal muscular atrophy (SMA) at the 66(th) American Academy of Neurology (AAN) meeting in Philadelphia, PA. http://photos.prnewswire.com/prnvar/20130807/LA60006LOGO Results from Phase 2 study in infants with SMA...

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...


Word of the Day
mallemaroking
  • Nautical, the visiting and carousing of sailors in the Greenland ships.
This word is apparently from a confusion of two similar Dutch words: 'mallemerok,' a foolish woman, and 'mallemok,' a name for some persons among the crew of a whaling vessel.