As part of the CZI Rare As One Network, patient-led organizations are developing and launching collaborative research networks in partnership with clinicians and scientists. The program provides funding, tools, and capacity-building support and training.
Nemaline myopathy (NM)
A Foundation Building Strength
A Foundation Building Strength is a nonprofit organization dedicated to finding treatments for Nemaline Myopathy (NM).
Cerebral cavernous angioma
Angioma Alliance works for those affected by cavernous angiomas by informing, supporting and empowering patients, and driving research for a cure.
Adult Polyglucosan Body Disease
APBD Research Foundation
APBD Research Foundation is dedicated to increasing public awareness and understanding of Adult Polyglucosan Body Disease and supporting research studies that may lead to new treatment options and a cure.
Cerebral Creatine Deficiency Syndromes
Association for Creatine Deficiencies
ACD provides patient, family and public education to advocate for early diagnosis and find medical research for treatments and cures for cerebral creatine deficiency syndromes.
The CACNA1A Foundation aims to accelerate the understanding, diagnosis and treatment of CACNA1A-linked diseases for today’s generation of patients.
Chelsea’s Hope Lafora Children Research Fund
Chelsea’s Hope Lafora Children Research Fund aims to fund research and improve the lives of those affected by Lafora disease.
CLOVES Syndrome & PIK3CA Related Overgrowth Spectrum (PROS)
CLOVES Syndrome Community
CLOVES Syndrome Community supports, educates, empowers, and improves the lives of those affected by CLOVES syndrome.
Congenital Hyperinsulinism International
Congenital Hyperinsulinism International is a lifeline to those born with congenital hyperinsulinism and their families.
Chronic Recurrent Multifocal Osteomyelitis
CRMO Foundation supports the health and well-being of patients and their families by furthering the efforts of life-changing research, increasing access to resources and education, and promoting awareness.
Congenital Muscular Dystrophy
Cure CMD is a nonprofit organization dedicated to curing Congenital Muscular Dystrophy by advancing research and empowering families.
Hereditary Hemorrhagic Telangiectasia
Cure HHT works to find a cure while saving the lives and improving the well-being of affected individuals and families.
CureGRIN is a parent-led foundation committed to improving the lives of people living with GRIN disorders.
LBSL (Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and Lactate Elevation)
Cure LBSL (formerly A Cure For Ellie) brings awareness, support, and hope to newly diagnosed families and individuals affected by rare disease LBSL.
Cure VCP Disease
Cure VCP Disease aims to drive efforts to cure diseases related to mutations of the Valosin Containing Protein gene
Deficiency of Adenosine Deaminase 2
DADA2 Foundation is a patient advocacy group supporting research for DADA2 and connecting families affected by the disease.
The DDX3X Foundation works to raise awareness, connect families, and find a cure for those with DDX3X syndrome.
FamilieSCN2A Foundation is an organization created by parents of children diagnosed with rare forms of Epilepsy and Autism as a result of a change in the SCN2A gene. Their vision is to find effective treatments and a cure for SCN2A-related disorders.
Fibrolamellar Hepatocellular Carcinoma
Fibrolamellar Cancer Foundation
The Fibrolamellar Cancer Foundation’s mission is to find a cure and treatment options, raise awareness of this disease, and connect and support the fibrolamellar community of patients and their families.
Foundation for Sarcoidosis Research
Foundation for Sarcoidosis Research is the leading international nonprofit organization dedicated to accelerating sarcoidosis research and providing support and education to those living with the disease.
Glut1 Deficiency Foundation
Glut1 Deficiency Foundation brings help and hope for the Glut1 Deficiency community.
Hermansky-Pudlak Syndrome Network
The Hermansky-Pudlak Syndrome Network is an advocacy organization for individuals and families affected by Hermansky-Pudlak syndrome.
Infantile Neuroaxonal Dystrophy
INADcure Foundation’s mission is to fund research that will lead to treatments and a cure for diseases caused by the impaired PLA2G6 gene.
The KAT6A Foundation supports the international KAT6A syndrome community.
KIF1A Associated Neurological Disorder
KIF1A.ORG is a global community dedicated to improving the lives of those affected by KIF1A Associated Neurological Disorder and accelerating research to find a cure.
Lennox-Gastaut Syndrome Foundation
The Lennox-Gastaut Syndrome Foundation's mission is to improve the lives of individuals affected by LGS through research, family support programs, and education.
Li-Fraumeni Syndrome Association
LFS Association provides a wide range of information, advocacy, and support services for individuals and families with Li-Fraumeni Syndrome.
Lymphangiomatosis & Gorham's Disease
Lymphangiomatosis & Gorham's Disease Alliance
Lymphangiomatosis & Gorham's Disease Alliance serves a worldwide community of patients, many of whom are young children, and their families.
Mission: Cure is a patient-led coalition of doctors, researchers, patients, and entrepreneurs pioneering a new approach to curing diseases, starting with pancreatitis.
The NEC Society is a collaborative organization dedicated to building a world without necrotizing enterocolitis.
PACS1 Syndrome Research Foundation
The PACS1 Syndrome Research Foundation supports research towards finding a cure for PACS1 Syndrome, a neuro-genetic disorder that causes significant intellectual disability, gross- and fine-motor delays, epilepsy, autism and severe speech delays.
Primary ciliary dyskinesia (PCD)
PCD Foundation is on a mission to improve the quality of life of those affected by PCD and to develop the means to find a cure.
Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network
PFIC Advocacy and Resource Network aims to improve the lives of patients and families worldwide affected by PFIC.
Chromosome 8p Disorders
Project 8p is driven by a simple question: What can we do for our child, family member, friend, or patient impacted by this diagnosis?
Primary Sclerosing Cholangitis
PSC Partners Seeking a Cure
PSC Partners Seeking a Cure provides education and support to primary sclerosing cholangitis patients, families, and caregivers and raises funds to research causes, treatments and cures.
Raymond A. Wood Foundation
The Raymond A. Wood Foundation advocates on behalf of hypothalamic-pituitary brain tumor patients, survivors and caregivers.
Recurrent Respiratory Papillomatosis (RRP)
Recurrent Respiratory Papillomatosis Foundation
The Recurrent Respiratory Papillomatosis Foundation helps educate and improve the lives of those affected by Recurrent Respiratory Papillomatosis.
Shwachman-Diamond Syndrome (SDS)
Shwachman-Diamond Syndrome Alliance
Shwachman-Diamond Syndrome Alliance is dedicated to building a world where Shwachman-Diamond Syndrome (SDS) is curable through driving research and engaging in advocacy and education.
Smith-Kingsmore Syndrome Foundation
The mission of the Smith-Kingsmore Syndrome Foundation is to improve the quality of life for children and families impacted by SKS by supporting cutting-edge research and collaboration among medical professionals, and strengthening community through worldwide awareness.
Systemic Juvenile Idiopathic Arthritis
Systemic JIA Foundation
Systemic JIA Foundation’s mission is to find a cure for Systemic Juvenile Idiopathic Arthritis, a sometimes lethal childhood disease.
TANGO2 Research Foundation
The TANGO2 Research Foundation’s mission is to improve the lives of children and young adults affected by the TANGO2 disease by helping to fund, coordinate, and guide scientific research.
Tatton Brown Rahman Syndrome (TBRS)
Tatton Brown Rahman Syndrome Community
The Tatton Brown Rahman Syndrome Community aims to support all families affected by TBRS and advance research toward interventions.
Dyskeratosis congenita/telomere biology disorders
Team Telomere supports those in the dyskeratosis congenita/telomere biology disorder community.
Citrate Transporter Disorder
TESS Research Foundation
TESS Research Foundation funds research in search of a treatment or cure for citrate transporter disorder, a genetic neurological disorder associated with the gene SLC13A5.
The Champ Foundation
The Champ Foundation supports research toward better treatment and a cure for Pearson Syndrome.
The EHE Foundation
The EHE Foundation’s goal is to connect its community to the latest research, treatment models, and events surrounding epithelioid hemangioendothelioma.
The Snyder-Robinson Foundation
The Snyder-Robinson Foundation is dedicated to furthering knowledge about Snyder-Robinson Syndrome through acquiring and sharing information with families, medical professionals, and researchers.
Stiff Person Syndrome
The Stiff Person Syndrome Research Foundation
The Stiff Person Syndrome Research Foundation is helping those affected by Stiff Person Syndrome by raising awareness and funds to find better treatments and a cure.
The TBCK Foundation
The TBCK Foundation works on behalf of the families impacted by TBCK Syndrome, which currently has around 90 known cases worldwide. It is a neuro-genetic disease causing epilepsy, intellectual and developmental disability, among other symptoms.
The Yaya Foundation for 4H Leukodystrophy
The Yaya Foundation for 4H Leukodystrophy fights for children with 4H Leukodystrophy by building an ecosystem of patients, families, researchers, clinicians, scientific experts, and supporters.
Usher Syndrome Type 1F
Usher 1F Collaborative
Usher 1F Collaborative funds medical research to find an effective treatment to save or restore the vision of those with Usher Syndrome type 1F.
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“By breaking down silos and encouraging parents, researchers and industry to collaborate, there is no stopping us.”
“Rare diseases have no better advocates than patients and caregivers.”
The Champ Foundation
“Science is always evolving, and that gives me hope.”