• Specifically Named Diseases

• 37.

Cardio Facio Cutaneous International

CFC International is dedicated to the support, research and treatment of individuals living with CFC Syndrome.

• International Development, Relief Services

• 38.

INTERNATIONAL PEDIATRIC TRANSPLANT ASSOCIATION INC

The International Pediatric Transplant Association (IPTA) is the only worldwide organization devoted to organ and tissue transplantation for children.

• Alliance/Advocacy Organizations

• 39.

PTEN Hamartoma Tumor Syndrome Foundation

PTEN is a tumor suppressor gene. When mutated at birth a patient is at higher risk for many #cancers and #autism.

Prader-Willi Syndrome Association | USA

What’s YOUR story. Tweet us and tell us what it’s like #GrowingUpRare We hope you will suggest the PWSA | USA facebook page to your friends.

• Management & Technical Assistance

• 41.

Pathways Corp

To elevate the patient's voice to increase access to care and eliminate bias by bringing stakeholders together Home Our Team Events Initiatives Partners Media Kit Contact More Its Time to Listen to the Rare Disease Community.

• Nerve, Muscle, and Bone Diseases

• 42.

CRMO FOUNDATION

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

• Biological, Life Science Research (includes Marine Biology, Physiology, Biochemistry, Genetics, Biotechnology, etc.)

• 43.

Cystic Fibrosis Foundation Therapeutics Inc

Recognized globally, the CF Foundation has led the way in the fight against cystic fibrosis, fueling extraordinary medical and scientific progress.

• Birth Defects, Genetic Diseases Research

• 44.

Channeling Hope Foundation

Channeling Hope Foundation A community of researchers and families driven to advance care and develop treatments for those affected by NALCNrelated disorders.

• Single Organization Support

• 45.

POMPE WARRIOR FOUNDATION

A non profit organization working to educate, research, and empower individuals and families affected by Pompe Disease and other rare diseases.