Patient Advocacy Groups h1 >
Batten Disease Support and Research Association
Determined to provide unwavering support, fund progressive research, and be a source of steadfast advocacy for all ceroid lipofuscinosis, neuronal (CLN) types.
Dana’s Angels Research Trust
Funds medical research, medical education, or medical or hospital care for the treatment or cure of Niemann-Pick Type C disease (NPC) or other similar genetic diseases.
Jonah's Just Begun - Foundation to Cure Sanfilippo, Inc
Raises and distributes funds to academic research groups focused on finding treatments for Sanfilippo syndrome, mucopolysaccharidosis (MPS) III.
National Gaucher Foundation
Serves U.S. patients with Gaucher disease and their families through financial support, education, patient services, and collaboration with medical professionals.
National Tay-Sachs & Allied Diseases Association, Inc.
Aims to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases by driving research, forging collaboration, and fostering community.
New Hope Research Foundation
Aims to provide hope to families and find a genetic cure for GM2 gangliosidosis (Tay-Sachs Disease) and other lysosomal storage diseases of the central nervous system.
Quebec Coalition of Orphan Diseases
Offrir un service d'information et de soutien pour les malades atteints d'une maladie rare et orpheline, leurs familles et pour les professionnels de la santé.
Rare Disorders NZ
Offers a starting point for patients and families affected by rare disorders, helping families, patients, and healthcare providers find essential information and support groups.