Links to Helpful Websites for Caregivers
and Healthcare Professionals
U.S. Government Resources
U.S. Food and Drug Administration
Learn more about information and view resources on clinical research studies.
National Institutes of Health
Read up on gene therapy in a comprehensive handbook on genetic research.
Patient Group Resources
The mission of ALD Connect is to improve health outcomes for individuals with X-linked adrenoleukodystrophy by raising awareness, empowering patients and accelerating the translation of scientific advances into better clinical care.
ALD Life is dedicated to providing support for patients and families with ALD and AMN. They provide practical support, advice and information, raise awareness of the diseases and advance medical research.
The Center for Information and Study on Clinical Research Participation
The Center for Information and Study on Clinical Research Participation (CISCRP) is a non-profit organization to educate and inform the public, patients, healthcare professionals, media and policy makers about clinical research.
The Global Genes Project
The non-profit Global Genes Project organization has been promoting the needs of the rare and genetic disease community since 2009 and has brought together support, knowledge and resources from more than 500 global organizations.
The Myelin Project
The Myelin Project is focused on improving the quality of life for those living with ALD and AMN through research, advocacy and family support.
National Organization for Rare Disorders
The National Organization for Rare Disorders (NORD) is a non-profit federation of voluntary health organizations dedicated to helping people with rare or orphan diseases. NORD is committed to identifying, treating and curing rare disorders through education, advocacy, research and service.
American Society of Gene and Cell Therapy
The American Society of Gene and Cell Therapy’s mission is to advance research and education leading to the discovery and application of gene and cell therapies. The society brings together stakeholders to incorporate the use of gene and cell therapies to alleviate human disease.
National Bone Marrow Donor Program
The National Bone Marrow Donor Program educates patients and families on marrow transplants for immune system and genetic disorders.
United Leukodystrophy Foundation
The United Leukodystrophy Foundation is dedicated to providing patients and their families with information about their disease and assistance in identifying sources of medical care, social services, and genetic counseling; establishing a communication network among families; increasing public awareness and acting as an information source for health care providers; and promoting and supporting research into causes, treatments, and prevention of the leukodystrophies.
World Leukodystrophy Alliance
The World Leukodystrophy Alliance is a group of like-minded non-profit organizations with the goal of leveraging resources to advance leukodystrophy treatment, care and research.