Gene Therapies - Success in treatment of SMA brings hope for children with rare diseases

The Research Australia (INSPIRE) winter issue features Gene Therapy success for infants with Spinal Muscular Atrophy (SMA).

After 25 years of incremental development the possibility of treating genetic diseases by repair or replacement of faulty genes is finally becoming a reality. A stunning example of this approach, known as gene therapy is the successful treatment of infants with SMA.

The SMA pilot program was supported and funded by Luminesce Alliance.