• Fund Raising and/or Fund Distribution

• 28.

Emily's Entourage

Accelerating lifesaving research and drug development for the final 10% of people with #cysticfibrosis that don't benefit from mutation-targeted therapies.

• Birth Defects, Genetic Diseases Research

• 29.

Ataxia Telangiectasia Children's Project, Inc.

The A-TCP organizes & supports innovative research to optimize disease management strategies, develop new treatments and find a cure for ataxia-telangiectasia.

• 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.

WIEDEMANN-STEINER SYNDROME FOUNDATION

The Wiedemann-Steiner Syndrome Foundation provides education, fosters community, and stimulates research to improve the lives of everyone impacted by #WSS.

• Specifically Named Diseases

• 32.

SETBP1 Society

President of SETBP1 Society and Parent Advocate for SETBP1-HD SETPB1 Society's mission is to provide support to individuals with SETBP1 haploinsufficiency disorde Our Mission is to provide support to individuals with SETBP1 disorder and their families, to promote discussion and fund research, and to bring awareness and education to the public.

• Nerve, Muscle, and Bone Diseases

• 33.

CRMO FOUNDATION

Supporting CRMO patients by furthering the efforts of life-changing research, increasing access to resources and education and promoting awareness.

Dlg4 Research Fund

DLG4-related Synaptopathy / SHINE Syndrome *Managed by the board of the DLG4 SHINE Foundation A page for people interested in learning more about DLG4-related Synaptopathy / SHINE Syndrome *Managed by the board of the DLG4 SHINE Foundation Mission To find specific treatment options and a cure for DLG4 patients by building a collaborative network of patients, families, clinicians and scientists that will work together to raise awareness and accelerate the understanding, diagnosis and treatment of DLG4 related synaptopathy.

• Fund Raising and/or Fund Distribution

• 35.

TEAM TREVOR

501c3 charity dedicated to advocation and eradication of cystic fibrosis.

• Fund Raising and/or Fund Distribution

• 36.

THE YAYA FOUNDATION FOR 4H LEUKODYSTOPHY

Fighting for children and families affected by 4H Leukodystrophy.