Cystinosis Research Foundation

The Cystinosis Research Foundation’s (CRF) mission is to find better treatments and a cure for cystinosis, a rare genetic disease that destroys every organ.

• Birth Defects, Genetic Diseases

• 20.

International Rett Syndrome Association, Inc.

We are transforming lives every day in our fight to treat and cure Rett syndrome and better the lives of families living with this devastating disorder.

• Birth Defects, Genetic Diseases Research

• 21.

Fam177a1 Research Fund

Founder and President, FAM177A1 Research Fund The mission of the FAM177A1 Research Fund is to improve the lives of those affected by FAM177A1 Associated Disorder through research and development of transformative therapies.

• Fund Raising and/or Fund Distribution

• 22.

Gardaccess Inc

Accelerating access to quality healthcare Addressing Unmet Needs in Rare Diseases Access to lifesaving medicine programs in low and middleincome countries LMIC presents a significant unmet need for patients living with rare disorders.

• Alliance/Advocacy Organizations

• 23.

Trpm3 Foundation

Patient advocacy and support for those affected by #TRPM3 #ionchannel related disorders.

• Alliance/Advocacy Organizations

• 24.

The Shank2 Foundation

To improve the quality of life for individuals affected by SHANK2 disorders.

• Fund Raising and/or Fund Distribution

• 25.

Tbc1D24 Foundation

Tbc1d24 foundation is a research organization and connecting site for caregivers, families, and those diagnosed with Tbc1d24 mutations.

• Birth Defects, Genetic Diseases Research

• 26.

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.

• Fund Raising and/or Fund Distribution

• 27.

ZZ One Family Foundation

The ZZ One Family Foundation exists to provide charitable fundraising support to community members and their families in times of extreme hardship, in hopes of providing healing and financial recovery.