Rare Conditions

Isis Pharmaceuticals Announces Phase 3 Study for Infants with SMA

Dosing with ISIS-SMN Rx is scheduled to begin within weeks.

U.S. House Passes Pediatric Research Network Bill

Support now needed for companion Senate bill.

Bill would fund research centering on rare illnesses and conditions in children.

Spinal Muscular Atrophy Gene Mutation Identified

Scientists at Cedars-Sinai Medical Center have uncovered a gene mutation responsible for spinal muscular atrophy with lower extremity predominance (SMA-LED).

Rare Diseases

Finding Someone Like Me

Understanding and Coping With the Challenges of Rare Diseases

Debra Richardson shares her story of finding a community for stiff person syndrome and how she started her own Web site to connect with others.

SMA Foundation Announces Biomarker Panel for SMA

A new biomarker assay panel for spinal muscular atrophy (SMA) could predict disease progression.