genetic disorders/rare disease Resources
RARE LIFE SOLUTIONS
From the small, hard-to-reach communities that define rare disease groups, require a different approach. We offer a broad suite of services that are designed to make drug development and commercialization better for industry and families.
22Q FAMILY Foundation
Dedicated to raising awareness for 22Q11.2 deletion syndrome while connecting, supporting, and offering accurate information to families affected by the disorder.
FOUNDATION FOR PRADER-WILLI RESEARCH
Through our world-class grants program, translational research programs, and PWS research tools, FPWR is playing an active role in research in order to develop new treatments and improve the health and well-being of those with PWS.
21 & YOU
We are a nonprofit dedicated to advocating, educating, and supporting families of children with Down syndrome. Our mission is to provide advocacy, educational resources, and communities of support that uplift and empower our community.
The warrior collective
The Warrior Collective is a non-profit organization dedicated to supporting individuals and their families living with rare disease. We provide for social, emotional and spiritual needs outside of medical treatment so they can enjoy a higher quality of life. At The Warrior Collective, we support the social, emotional, and spiritual needs of those 25 to 30 million Americans and their families so they can enjoy a higher quality of life.
National organization for rare disorders
The mission of NORD is to improve the health and well-being of people with rare diseases by driving advances in care, research and policy.
NATIONAL Niemann-Pick DISEASE FOUNDATION
The NNPDF is a non-profit, patient advocacy and family support organization dedicated to supporting and empowering patients and families affected by Niemann-Pick disease, through education, collaboration and research.
CURE SANFILLIPPO Foundation
Cure Sanfilippo Foundation architects and funds cutting-edge research to accelerate discovery of a treatment or cure for Sanfilippo Syndrome so parents can dream of their children’s futures again.
WILLIAMS SYNDROME ASSOCIATION
The WSA works to advance the interests of all individuals with Williams syndrome throughout their lifespan by providing programming and resources, supporting research, promoting partnerships and connections.
NATIONAL DOWN SYNDROME SOCIETY
NDSS empowers individuals with Down syndrome and their families by driving policy change, providing resources, engaging with local communities, and shifting public perceptions.
Dream factory INC
A 501(c)(3) nonprofit and the largest all-volunteer driven children’s wish-granting organization that does not limit its mission to children who have life-threatening illness. The organization believes children with chronic illnesses and disorders also suffer from substantial emotional and physical pain.
STAR Foundation
Salla Disease is the most prevalent Free Sialic Acid Storage Disorder (FSASD), a family of genetic diseases caused by the accumulation of the natural sugar, sialic acid. The disease primarily impacts the central nervous system causing serious developmental and cognitive delays. 23,000 patients worldwide are estimated to be affected by the disease but due to poor diagnosis only about 300 cases have been reported.
Australian Niemann-Pick DISEASE FOUNDATION
The Australian NPC Disease Foundation (ANPDF) aims to give hope, support and connection to those affected by Niemann-Pick disease type C (NPC) and Acid Sphingomyelinase Deficiency (ASMD) in Australia.
NATIONAL FRAGILE X FOUNDATION
Since 1984, the NFXF has supported and empowered families impacted by Fragile X by providing the knowledge, resources, and tools they need to navigate daily challenges and support individuals in reaching their full potential.
TANGO 2 research
TANGO2 Research Foundation hopes that by helping to fund, coordinate and guide the efforts of researchers, scientists and doctors, we can rapidly accelerate the understanding around TANGO2 and pave the way to future therapies and positive outcomes for loved ones.
GLOBAL DOWN SYNDROME FOUNDATION
The Global Down Syndrome Foundation is a public non-profit 501(c)(3) dedicated to significantly improving the lives of people with Down syndrome through Research, Medical care, Education and Advocacy.
Sunshine foundation
Sunshine Foundation is a unique children’s charity, as it is the only national wish-granting organization that answers the dreams or wishes of children with life-long chronic illnesses or conditions, including level 3 severe autism and Down syndrome. Unlike other charities that typically turn away children because their diagnosis is not considered life-threatening or critical, Sunshine Foundation is proud to serve these deserving children and their income-limited families.