ASGCT (American Society of Gene & Cell Therapy)
A professional membership organization for scientists, physicians, patient advocates, and other professionals in gene and cell therapy, working in settings such as universities, hospitals, and biotechnology companies. Provides information on the science, technology, and use of gene and cell therapy as well as a Patient Education Program.
Cure SMA
An advocacy organization that funds treatment research and provides support for individuals with spinal muscular atrophy (SMA) and their families. Their site includes detailed information and resources on the various types of SMA and the current and future genetic research planned for this rare disease for the newly diagnosed, caregivers, healthcare providers, researchers, and fundraisers.
NORD (National Organization for Rare Disorders)
NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. Their site includes information about opportunities to participate in clinical trials and other studies so that patients and their physicians may decide whether specific studies are appropriate for them.
