At Novartis Gene Therapies，we are as passionate about out patients as we are about research.We are dedicated to supporting the communities and families affected by rare genetic diseases， and committed to our continued pursuit of reimaging solutions for the significant and unique challenges that life with a rare genetic disease can bring.That's why we strive to be 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 proudly share our resources and voice to activities that benefit families impacted by rare disease。

上下支撑，上下支撑

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

已修复和已修复区域：

  • Cure SMAprovides support to patients and families affected by SMA and funds and directs research for a cure for the disease。
  • Global Geneshelps build awareness，educate the global community，and provide critical connections and resources that equip advocates to become activists for their rare disease。
  • uscular Dystrophy Associationsponsors research seeking causes and effective treatments for neuromuscular diseases。
  • National Organization for Rare Disorders（NORD）provides support for individuals with rare diseases by advocating and funding research，education，and networking among service providers。
  • NEVER GIVE UPis dedicated to raising global SMA awareness，advocating for research，and providing family support。
  • RareConnectis a platform with disease-specific online communities that enables people living with rare diseases to meet，share stories and learn from each other。
  • SMA Foundationfunds research to accelerate the development of treatments for SMA
  • SMA News Todayprovides SMA news，education，and occasional insights about clinical trials in the SMA research community。
  • 雷尔置换为The EveryLife Foundation for Rare Diseasesis dedicated to accelerating biotech innovation forrare disease treatment through science-driven public policy。
  • Treat-NMDprovides a platform to ensure the most promising new therapies in the neuromuscular field reach patients as quickly as possible。