• Birth Defects, Genetic Diseases Research

• 37.

SMS Research Foundation

The mission of the Smith-Magenis Syndrome Research Foundation is to advance scientific research leading to innovative treatment options for people living with SMS.

The Global Foundation for Peroxisomal Disorders

The GFPD's mission is to improve the lives of individuals with peroxisomal disorders by funding research, championing scientific collaboration, and empowering families and professionals through educational programs and support services.

THE CLINIC FOR SPECIAL CHILDREN INC

A nonprofit comprehensive medical practice focused on treating & researching rare genetic disorders.

Retina Foundation of the Southwest

We are a 501(c)(3) nonprofit located in Dallas, TX with the mission to prevent vision loss and restore sight through innovative research and treatment.

• Birth Defects, Genetic Diseases Research

• 41.

Charleys Fund Inc

Our goal is to cure Duchenne Muscular Dystrophy in time to save Charley's life and the lives of thousands of boys like him.

• Birth Defects, Genetic Diseases

• 42.

Stop ALD Foundation

The Stop ALD Foundation: Adrenoleukodystrophy (ALD) therapy development, awareness, & prevention.

• Epilepsy Research

• 43.

Bridge the Gap

SYNGAP1 Foundation is a legacy page dedicated to preserving the work and mission of our 100% charitable nonprofit in the USA, focused on #SYNGAP1 To serve, educate and fund research for families coping with the effects of SYNGAP mutations.

The RYR-1 Foundation

This organization is to raise awareness, educate, provide support, and raise funds for research.

• Birth Defects, Genetic Diseases

• 45.

PKS Kids

Hope and help for those touched by Pallister-Killian Syndrome PKS is a rare tetrasomy of 12p.