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 04:25

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



PTEN Depletion Found To Decrease Spinal Muscular Atrophy Disease Severity ...
Spinal muscular atrophy (SMA) is an autosomal recessive disorder that causes loss of motor neurons and atrophy of the muscle, with patients presenting loss of strength in proximal muscles. SMA is caused by mutations or deletion of the telomeric copy of ...
SMA News Today - Fri, 19 Dec 2014 11:18



Spinal Muscular Atrophy Study in Mice Reveals Deleting Atrophy Enhancing ...
Spinal muscular atrophy (SMA) is an autosomal recessive disorder that causes loss of motor neurons and atrophy of the muscle, with patients presenting loss of strength in proximal muscles. It has been suggested that preventing muscle atrophy could ...
SMA News Today - Tue, 16 Dec 2014 07:33



AveXis Launches Spinal Muscular Atrophy Clinical Trials Website
AveXis Dallas, Texas-based AveXis, Inc., a biotechnology company working to improve the quality of life of patients suffering from severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA) through the use of gene therapy, is launching a new ...
SMA News Today - Thu, 04 Dec 2014 08:37

AveXis Announces Launch of New Patient-Oriented Website Focused on Spinal ...
PR.com (press release) - Thu, 04 Dec 2014 00:08

Benefit for Isaiah set for Dec. 7
Newrichlandstar - Thu, 04 Dec 2014 08:30



Phase 3 Spinal Muscular Atrophy Study Begins For Experimental ISIS-SMN Rx ...
Spinal Muscular Atrophy (SMA) is a disease caused by mutations in the Smn1 gene that encodes SMN1 protein, a key protein for motor neuron survival. The lack of SMN protein leads to motor neuron dysfunction and death, triggering subsequent generalized ...
SMA News Today - Fri, 28 Nov 2014 13:00



Dosing Completed For Type 1 Spinal Muscular Atrophy Clinical Trial
therapy for Type 1 SMA Orphan drug development company AveXis Inc. recently announced the completion of the dosing phase in the low-dose cohort of the company's ongoing clinical trial efforts for its experimental Type 1 spinal muscular atrophy (SMA) ...
SMA News Today - Fri, 05 Dec 2014 08:21



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



Parents Juieanne Bugeja and Chris Kotsopoulos with Alyssa, who needs a new ...
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 04:56


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Last update: September 2014