Congratulations to the 2021 Health Equity RARE Patient Impact Grant Winners!
The Mission of the ALD Alliance is to:
- Advocate for ALD and all RUSP Newborn Screening conditions Nationally
- To provide financial support to families affected
- To fund research efforts for ALD
- To raise awareness of ALD
Through the efforts of the foundation: “Aidan’s Law” was signed on March 29, 2013. “Aidan’s Law” requires every baby born in New York State to be tested for ALD at birth. New York State was the first in the country and in the world to test newborns for ALD. Our work continues in the states where we are now at 23 states testing for ALD. We have expanded our efforts to fight for all newborn screening conditions added to the Federal Recommended Uniform Screening Panel and to ensure EVERY baby born in the USA has the same chance at a normal, healthy life.
Angioma Alliance is the only United States patient research organization for those affected by cerebral cavernous malformations (cavernous angioma, cavernoma). Our mission is to inform, support, and mobilize those affected by cerebral cavernous malformations and drive research for better treatments and cure. Our programs include an annual international scientific meeting, a patient registry and DNA/Tissue bank, free genetic testing, clinical trial consultation and support, outreach to underserved and special populations, recognized CCM Centers of Excellence, and clinical care consensus guidelines. We offer numerous venues for patient support and education and mentor international CCM patient organizations.
The Bleeding Disorders Alliance Illinois (BDAI) supports families affected by inherited bleeding disorders such as Hemophilia and von Willebrand disease by providing its members with the unbiased medical information and resources that allow them to live a healthy and active lifestyle.
These rare diseases, which affect about 1% of the general population, are often difficult to diagnose and are two of the most costly chronic disorders to treat. The ability to access reliable and unbiased information is especially important for families seeking answers and support soon after the initial diagnosis. BDAI serves as a non-profit connection between its members and the many medical providers, treatment centers, public and private insurance agencies and government programs that offer medical and financial assistance.
The organization also offers a welcoming community for families to meet, interact and share information and advice. By providing popular social and education events for youth, families and parents throughout the year, BDAI helps foster a sense of connection between its members and provides continual opportunities to learn new ways to manage life with these disorders.
Through education and support, BDAI helps its members advocate on their own behalf with public and private insurance providers, medical providers, and government agencies while representing the bleeding community in the world at large.
The Bleeding Disorders Alliance Illinois believes that, with the proper support and resources, its members have the potential to be defined not by their conditions, but by their contributions to the world.
CFC International has dedicated 2 decades to improving the lives of individuals with CFC syndrome and their families through family support, research, and education. With more than 800 individuals diagnosed with CFC syndrome, our vision is to advance research and improve treatment. CFC syndrome is a rare genetic disorder often diagnosed in early childhood that occurs due to a mutation in one of 5 genes: BRAF, KRAS, Map2k1, Map2k2, or YWHAZ. The disease causes neurological, cardiological, and dermatological complications – among many other comorbidities – that are often life threatening.
Foundation for Sarcoidosis Research
The Foundation for Sarcoidosis Research is the leading international organization dedicated to finding a cure for sarcoidosis and to improving care for sarcoidosis patients. Since its establishment in 2000, FSR has fostered over $5 million in sarcoidosis-specific research efforts. Sarcoidosis is a rare inflammatory disease that causes cells to form clumps called granulomas. When too many granulomas form on an organ, they can interfere with the organ’s function. Sarcoidosis can affect any organ in the body; however, 90 percent of cases impact the lungs. For more information about FSR visit: stopsarcoidosis.org.
National PKU News
National PKU News provides resources and support for individuals, families, and clinicians managing PKU (Phenylketonuria) and other inborn errors of metabolism. Our mission is to leverage innovation, insight, and research to improve the health, well-being, and daily lives of those with PKU and other IEMs.
Sick Cells is a patient advocacy organization that seeks to elevate the voices of the sickle cell disease (SCD) community and our stories of resilience. The mission of Sick Cells is to improve the care of and quality of life for people living with SCD, including ending the stigma of SCD, by shaping public policy and educating the SCD community to be engaged advocates, thus creating greater awareness in the general public overall. To these ends, Sick Cells is working to (1) Elevate the voices of the patients, caregivers, and the entire SCD community, (2) Educate decision-makers about the grave challenges, unique needs, and sharp disparities this community faces, and (3) Unite and empower the SCD community to effectively advocate on important topics and policies.
The Sickle Cell Association of Texas Marc Thomas Foundation
The Sickle Cell Association of Texas Marc Thomas Foundation provides numerous services to children, adults, and families in Central Texas, San Antonio, the Texas Gulf Coast, and other areas affected with sickle cell disease and those carrying sickle cell trait. We provide education, research awareness, outreach, support group meetings, assistance, and numerous other services.
The Akari Foundation
Ariel and Enrique Najera the reason behind The Akari Foundation. 11 yrs old fraternal twins, both diagnosed with Duchenne Muscular Dystrophy since 2016, since my journey in search of the best possibilities for my former partner boys, we noticed a lack of information and support to low-income families and language barriers. With my background in the marketing and entertainment field, I am convinced I can create a Therapy Holistic and education advocacy center for the Hispanic immigrant and low-income families.
Uplifting Athletes, a nonprofit organization founded in 2007, inspires the Rare Disease Community with hope through the power of sport. Since 2007, Uplifting Athletes has raised more than $6 Million to develop Uplifting Leaders, create Uplifting Experiences, fund rare disease research and expand rare disease awareness.