Isis starts Phase 3 trial in spinal muscular atrophy
Isis Pharmaceuticals has started a second Phase 3 study of its drug for spinal muscular atrophy, a severe genetic disease that can be fatal. In connection with the study, the Carlsbad biotech said Tuesday it has earned a $27 million milestone payment ...
U-T San Diego - Tue, 25 Nov 2014 18:18

Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Children With ...
MarketWatch - Tue, 25 Nov 2014 04:03

Isis Pharma (ISIS) Initiates Pivotal ISIS-SMNRx Phase 3
StreetInsider.com (subscription) - Tue, 25 Nov 2014 04:00

Isis Pharma starts Phase 3 SMA trial
Seeking Alpha - Tue, 25 Nov 2014 05:30

Combating spinal muscular atrophy
SMA is a genetic disorder that can rob people of strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat or breathe. Miller-Smith and Fiorenna Fuentes-Israel, who also co-founded the local Cure SMA chapter ...
Sun Sentinel - Wed, 05 Nov 2014 15:08

CSF-Delivered Gene Therapy Shows Promise for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is caused by a mutation in the survival motor neuron 1 gene (SMN1). Because of this mutation, the individual does not produce enough survival motor neuron (SMN) protein. Gene transfer may increase SMN levels by using a ...
PR Newswire (press release) - Wed, 05 Nov 2014 13:00



Brave child needs a new wheelchair
But unlike most other children, the young St Albans girl's number one wish is for a new wheelchair. When she was nine months old, Alyssa was diagnosed with Spinal Muscular Atrophy, which affects motor nerve cells and takes away the ability to walk.
Herald Sun - Sun, 23 Nov 2014 05:04

Spinal Muscular Atrophy Program Enters Phase 1b/2a Trial In Patients With SMA
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a Phase 1b/2a study in adult and pediatric patients.
Benzinga - Wed, 19 Nov 2014 04:56

Can Kaden be cured? West Palm Beach parents spread awareness about fatal ...
West Palm Beach couple Katie and Kyle Myers with their 10-month-old son, Kaden, on the beach. Kaden suffers from the often-fatal genetic disease, spinal muscular atrophy (SMA). (Photo by Strictly Bella Photography). Designed for readers. Exclusively ...
Palm Beach Post - Mon, 17 Nov 2014 15:53



PTC's Spinal Muscular Atrophy Phase 1b/2a Trial Begins
shutterstock_31035061 PTC Therapeutics, Inc. announced that their collaborative program with Roche and the SMA Foundation (SMAF) for Spinal Muscular Atrophy (SMA) has begun its second phase with adult and children patients, as part of the Phase ...
SMA News Today - Fri, 21 Nov 2014 17:30



Area family recruits bands for Spinal Muscular Atrophy benefit
Adyn Bucher, 10, and her family are hosting a benefit concert at Jackie O's Public House on Saturday night to raise funds for Adyn's Dream — a nonprofit that benefits families that have members who are living with Spinal Muscular Atrophy. Adyn was ...
Athens Messenger - Wed, 29 Oct 2014 21:30


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Last update: September 2014