Isis initates phase I spinal muscular atrophy study

Monday, December 19, 2011 11:34 AM

Isis Pharmaceuticals has initiated a phase I study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA).  SMA is a severe motor-neuron disease that is the leading genetic cause of infant mortality. Isis is developing ISIS-SMNRx as a potential treatment for all types of SMA.

The phase I study of ISIS-SMNRx is a single-dose, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of the drug in children with SMA between the ages of 2-14 who are medically stable.  In this study, ISIS-SMNRx will be administered intrathecally as a single injection directly into the spinal fluid. Intrathecal administration of an antisense drug, ISIS-SOD1Rx, has been shown to be safe and well tolerated in an ongoing phase I study in patients with amyotrophic lateral sclerosis.

Share:          
CWWeekly

May 18

Research for All Act reaches Congress for second time seeking gender equality in basic research, clinical trials

MediciGlobal finds fewer trial dropouts among participants who actively pursue enrollment versus those recruited

Already a subscriber?
Log in to your digital subscription.

Subscribe to CWWeekly.

The CenterWatch Monthly

May

Job satisfaction mixed as workload increases
Salaries not keeping pace with rise in work, responsibilities

Gamification moving from early science to patient use
Pharma looking to use games for early diagnosis, recruitment, adherence

Already a subscriber?
Log in to your digital subscription.

Purchase the May issue.

Subscribe to
The CenterWatch Monthly.

The CenterWatch Monthly

April

PSI, INC Research, Chiltern best with sites
Strategic outsourcing prioritizing, improving site relationships

Regulatory compliance an increasing burden on sites
Increased staff time, materials, storage eroding site operating profit

Already a subscriber?
Log in to your digital subscription.

Purchase the April issue.

Subscribe to
The CenterWatch Monthly.

JobWatch centerwatch.com/jobwatch

Featured Jobs