Resources for the Rare Disorders Patient Community

At Novartis Gene Therapies, we are as passionate about our patients as we are about our research. We are dedicated to our commitment to the communities and families affected by the conditions we are studying. We know that life with a rare genetic disease can bring significant challenges, and we want to be as supportive as possible. That’s why we strive to act as an ally and resource to the communities affected by the conditions we study.

Our efforts to help patients and caregivers

Novartis Gene Therapies has worked with leading rare disease advocacy organizations on matters of policy and patient support. We gladly lend our resources and voice to activities that benefit families impacted by rare disease.

Ongoing support of communities

Whether it is an educational presentation, a workgroup to better understand the patient journey, or participation in an event, we are honored to be an active ally in the communities we serve.

The following organizations offer resources and information for patients and loved ones impacted by rare disorders:

  • Global Genes - Started under a unifying symbol of hope—the Blue Denim Genes Ribbon—this organization helps build awareness, educate the global community, and provide critical connections and resources that equip advocates to become activists for their rare disease
  • International Rett Syndrome Foundation - Their mission is to accelerate full spectrum research to cure Rett syndrome and empower families with information, knowledge, and connectivity
  • Muscular Dystrophy Association - The world’s leading nonprofit health organization sponsoring research seeking causes and effective treatments for neuromuscular diseases
  • Rett Syndrome Research Trust - RSRT was launched in 2008 to drive research toward a cure for Rett Syndrome and related MECP2 disorders. Their mission is urgent: 15,000 girls and women in the United States and 350,000 globally live with the agonizing symptoms of Rett syndrome
  • SMA Foundation - Established by Loren Eng and Dinakar Singh, parents of a child with SMA, to accelerate the development of treatment as the leading SMA research funder
  • SMA News Today - This science and health web publication provides SMA news, education, and occasional insights about clinical trials in the SMA research community
  • The EveryLife Foundation for Rare Diseases - A foundation for rare diseases, dedicated to accelerating biotech innovation for rare disease treatment through science-driven public policy
  • The Gwendolyn Strong Foundation - Born from the Strong family’s firsthand experience with SMA Type 1, they are dedicated to increasing global SMA awareness, research, and family support
  • Treat-NMD - A network for the neuromuscular field that provides an infrastructure to ensure the most promising new therapies reach patients as quickly as possible