• Epilepsy

• 28.

Where Theres A Will Theres A Cure

We are going to cure X-Linked Myotubular Myopathy.

Rare Trait Hope Fund

Rare trait Hope Fund facilitates research and provides funding for the development of treatments for a rare disease called Aspartylglucosaminuria (AGU) in hopes to save lives of AGU kids and adults.

• Birth Defects, Genetic Diseases Research

• 30.

Spata Foundation Inc

Our mission is to advocate, educate, and raise funds for research towards SPATA5 & SPATA5L1 Disorders The SPATA Foundation's Mission is to advocate, educate, and drive research towards SPATA5 and SPATA5L1 Related Disorders.

• Nerve, Muscle, and Bone Diseases

• 31.

The Lilly and Blair Foundation

The Lilly and Blair Foundation is dedicated to improving the lives of children battling de novo spastic paraplegia - a swift and severe neurodegenerative disease - through collaboration with top scientists and funding of promising research.

• Diseases, Disorders, Medical Disciplines N.E.C.

• 32.

Rare Genes Movement

Our vision is to see every child have access to proper medical, therapeutic, and educational resourc Our purpose is to raise awareness and funds to help families of children with rare genetic disorder.

• Specifically Named Diseases

• 33.

Sisters Hope Foundation for Hereditary Diffuse Leukoencephalop

We are dedicated to amplifying patient voices, and one day meeting the first survivor of ALSP.

United MSD Foundation

Our mission is simple, yet powerful: To cure Multiple Sulfatase Deficiency.

• Alliance/Advocacy Organizations

• 35.

Rare Disease Company Coalition Inc

We are a coalition of life science companies committed to discovering, developing & delivering rare disease treatments for the patients we serve.

• Fund Raising and/or Fund Distribution

• 36.

THE YAYA FOUNDATION FOR 4H LEUKODYSTOPHY

Fighting for children and families affected by 4H Leukodystrophy.