Dosing with ISIS-SMN Rx is scheduled to begin within weeks.
Bill would fund research centering on rare illnesses and conditions in children.
Scientists at Cedars-Sinai Medical Center have uncovered a gene mutation responsible for spinal muscular atrophy with lower extremity predominance (SMA-LED).
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.
A new biomarker assay panel for spinal muscular atrophy (SMA) could predict disease progression.