A Committed partner

At AveXis, we’re as passionate about our patients as we are about our research. That’s why we strive to act as an ally and resource to the communities affected by the conditions we study.

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

ALS Association

Established in 1985, the ALS Association is the only national nonprofit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, the ALS Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.

ALS Worldwide

ALS Worldwide provides FREE personalized and confidential support via videoconference, email, social media, phone, and in-person visits to people living with ALS in more than 135 countries.

Check Orphan

This nonprofit organization is dedicated to people working with or affected by rare, orphan, or neglected diseases.

Cure SMA

Dedicated to the treatment and cure of spinal muscular atrophy (SMA), they fund groundbreaking research and provide families the support they need for today.

Girl Power 2 Cure

Founded in 2006, Girl Power 2 Cure is a dedicated and vibrant nonprofit organization leading a movement to end Rett Syndrome, a severe neurological disorder that primarily affects girls. They are here to respond to the urgent need for support, education, mainstream awareness, and treatments.

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.

National Institute of Neurological Disorders and Stroke

NINDS seeks fundamental knowledge about the brain and nervous system in order to reduce the burden of neurological disease.

National Organization for Rare Disorders

This national advocacy organization for rare diseases is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.

Project ALS

Since it was founded in 1998, Project ALS has identified and funded scientific and medical research with the goal of leading to the first effective treatments and cure for ALS. The group recruits the world’s best scientists and doctors to work together to develop a better understanding of the ALS disease process and, in parallel, better therapeutic strategies.

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.

The Sophia’s Cure Foundation

Formed by Catherine and Vincent Gaynor, shortly after their daughter was diagnosed with SMA, to assist in clinical research funding and family support.


A network for the neuromuscular field that provides an infrastructure to ensure the most promising new therapies reach patients as quickly as possible.