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Resources
Managed Access Program
Patients with serious or life-threatening diseases or conditions sometimes seek medical products that are not yet approved or available in their country. Novartis Gene Therapies’ “Managed Access” addresses this need by making certain investigational or unapproved treatments available to eligible patients. Click here to learn more.
Clinical Trial Information
ClinicalTrials.gov
Clinicaltrials.gov is a database of privately and publicly funded clinical studies conducted around the world.
StudySMAnow.com
Learn about Novartis Gene Therapies-sponsored research studies for spinal muscular atrophy (SMA).
Neuromuscular Disease Resources
SMA Europe
SMA Europe is an umbrella organisation, which includes Spinal Muscular Atrophy (SMA) patient and research organisations from across Europe.SMA Europe campaigns to improve the quality of life of people who live with SMA.
TREAT-NMD.eu
TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.
MDA.org
MDA works to find research breakthroughs across diseases, provides support for individuals from day one at MDA Care Centers, and empowers families with services and support in hometowns across America.
SMAnewstoday.com
Spinal Muscular Atrophy News Today provides news and information about spinal muscular atrophy.
NORD (RareDiseases.org)
NORD (National Organization for Rare Disorders) is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.