BEYOND BATTEN DISEASE FOUNDATION

Mission: eradicate Batten disease. Batten disease is a fatal neurodegenerative disorder that takes away childhood, and then takes away.

Charcot-Marie-Tooth Association

CMT is the most commonly inherited peripheral neuropathy.

• Nerve, Muscle, and Bone Diseases

• 93.

Reflex Sympathetic Dystrophy Syndrome Association (RSDSA)

RSDSA's mission is to provide support, education, and hope to everyone affected by #CRPS / #RSD while we drive research to develop better treatment and a cure.

• Birth Defects, Genetic Diseases

• 94.

Gene Spotlight Inc

We are a nonprofit medical research and advocacy organization committed to spark breakthroughs in treatments and cures for rare genetic disorders, including lysosomal storage diseases, uncommon cancers and other orphan diseases.

Alpha-1 Foundation, Inc.

Our mission is to increase awareness of and find a cure for Alpha-1 Antitrypsin Deficiency.

• Specifically Named Diseases Research

• 96.

PROJECT ALIVE

A Hunter syndrome research and advocacy foundation.

Kcnq2 Cure Alliance Inc

Our promise is to educate and to advance research leading to treatments or a cure for patients living with the genetic disorder KCNQ2 Raising awareness, educating, and advancing research for KCNQ2 Developmental and Epileptic Encephalopathy.

COPA Syndrome Foundation

The COPA syndrome is an inherited disorder that is caused by mutations in the COPA gene.

• Birth Defects, Genetic Diseases

• 99.

Louies Huwe

Looking for information on HUWE1 gene variants.