Additional Resources

National Organization for Rare Diseases Logo

The National Organization for Rare Disorders

The National Organization for Rare Disorders (NORD®), an independent nonprofit, is leading the fight to improve the lives of rare disease patients and families. We work together to accelerate research, raise awareness, provide valuable information and drive public policy that benefits the estimated 25-30 million Americans impacted by rare diseases.
Global Genes Logo

Global Genes

Global Genes is an organization that helps to build awareness, educate the global community, and provide critical connections and resources that equip advocates to become activists for their rare diseases.
Child Neurology Foundation Logo

Child Neurology Foundation

The Child Neurology Foundation connects partners from all areas of the child neurology community so those navigating the journey of disease diagnosis, management, and care have the ongoing support of those dedicated to treatments and cures.
Everylife Foundation Logo

EveryLife Foundation

EveryLife Foundation is dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy.
Eurordis Logo


EURORDIS-Rare Diseases Europe is a not-for- profit alliance of 1000 rare disease patient organisations from over 70 countries around the world that work together to improve the lives of the 30 million Europeans living with a rare disease. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies and patient services.
Rare Diseases Clinical Research Network Logo

Rare Disease Network

Rare Diseases Clinical Research Network is a group committed to advancing medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment, and data sharing.
Genetic Alliance Logo

Genetic Alliance

Genetic Alliance is an advocacy organization that engages individuals, families, and communities to transform health.
Alliance for Regenerative Medicine Logo

Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine is an international community of small and large companies, non-profit research institutions, patient organizations, and other sector stakeholders dedicated to realizing the promise of regenerative medicine for patients around the world.
ARM Foundation for Cell & Gene Medicine Logo

ARM Foundation

The ARM Foundation for Cell and Gene Medicine educates, engages, and empowers patients, caregivers, industry leaders, and other stakeholders to help advance the science and benefits of gene and cell therapy.
American Society of Gene and Cell Therapy Logo

American Society of Gene and Cell Therapy (ASGCT)

The American Society of Gene and Cell Therapy (ASGCT) is a professional membership organization for scientists, physicians, advocates, and other professionals in gene and cell therapy. A goal of the Society is to provide timely, accurate, and responsible information about gene and cell therapy to patients and their families through the Patient Education website, including resources on gene therapy for Batten disease.
National Alliance for Caregiving Logo

National Alliance for Caregiving

Established in 1996, the National Alliance for Caregiving is a non-profit coalition of national organizations focusing on advancing family caregiving through research, innovation, and advocacy. The Alliance conducts research, does policy analysis, develops national best-practice programs, and works to increase public awareness of family caregiving issues. Recognizing that family caregivers provide important societal and financial contributions toward maintaining the well-being of those they care for, the Alliance supports a network of more than 80 state and local caregiving coalitions and serves as Founder and Secretariat for the International Alliance of Carer Organizations (IACO).
Courageous Parents Network’s Logo

Courageous Parents Network’s (CPN)

Courageous Parents Network’s (CPN) mission is to empower, support and equip families and providers caring for children with serious illness. CPN’s vision promotes the family’s journey as one in which they have confidence in their ability to be the best caregivers they can possibly be, resulting in minimal regret and maximal healing. CPN resources include 500+ original short videos, a blog, curated content modules (Guided Pathways) and downloadable guides on topics spanning the wide range of issues including coping with the diagnosis, understanding anticipatory grief, working with the medical team, making difficult decisions about medical interventions and coping following loss. Much of this content is also offered in Spanish.
The Mighty Logo

The Mighty

The Mighty is a supportive health community for people facing challenges and their families.