Foundation for Angelman Syndrome Therapeutics

FAST is the premier patient advocacy organization working to cure Angelman syndrome.

THE CLINIC FOR SPECIAL CHILDREN INC

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

• Research Institutes and/or Public Policy Analysis

• 75.

Forgotten Diseases Research Foundation

THE FORGOTTEN DISEASES RESEARCH FOUNDATION IS A NONPROFIT DEDICATED TO VERY RARE DISORDERS.

• Specifically Named Diseases

• 76.

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.

• Fund Raising and/or Fund Distribution

• 77.

Life for Liam and Friends Foundation

Mission to support families who are impacted by NPC and help find a cure through connecting NPC families, spreading awareness, and supporting medical research.

• Specifically Named Diseases

• 78.

YBRP Inc

YBRP is a non-profit organization whose mission is to positively impact the lives of individuals with HNRNPH2 genetic variations.

• Fund Raising and/or Fund Distribution

• 79.

THE CHAMP FOUNDATION

Mission The Champ Foundation supports research toward better treatment and a cure for single largescale mitochondrial deletion syndromes SLSMDS like Pearson syndrome.

• Fund Raising and/or Fund Distribution

• 80.

TO CURE A ROSE FOUNDATION

At To Cure A Rose Foundation, we’re on a mission to make genetic treatments faster, more affordable, and within reach—so that the next child born with a rare disease can receive a customized drug within days of diagnosis.

• Specifically Named Diseases Research

• 81.

Dion Foundation for Children with Rare Diseases Inc

Support research. Find a cure. Help children and families fight rare diseases.