Alport Syndrome Foundation
Alport Syndrome Foundation is honored to announce that Dr. Moumita Barua and Dr. Andrew Paterson have received the 2020 Research Award of $125,000 over two years, made possible by the generosity of patients, families and friends. Learn about their project to help answer questions about disease progression patterns related to specific sex and genetic mutation. More.
Amyloidosis
Foundation
Amyloidosis Foundation invites you to join their virtual wellness challenge through October 31 in collaboration with Kilter Rewards to support and raise essential funding for the Foundation. Participants earn points every day for making healthy choices and compete for cash prizes. This event is for all fitness levels! More.
Child and Youth Care
Child and Youth Care is excited to
share their new website to better defend, champion and advance the interests of children living with rare bleeding disorders to improve the quality of life in Zimbabwe. More.
CMTC-OVM
CMTC-OVM is proud to support patients and families holistically. All are welcome to look at their new brochures in multiple languages, not only about CMTC, but also about disease-independent topics including psychology, conversations with health care providers and support for siblings. More.
The global CMTC-OVM member conference on October 24 will now be a virtual event. Review the agenda and register today! More.
Cornelia de Lange Syndrome Foundation
Join Cornelia de Lange Syndrome Foundation for the biennial Scientific and
Educational Symposium on October 2-3. This event brings together researchers to present current and new information related to CdLS and broaden their collective knowledge. Learn about medical and educational issues facing individuals living with the syndrome and an update on scientific advances. More.
HCU Network America
HCU Network America and Organic Acidemia Association are excited to co-host the Land of the Free, Home of the Brave, 2021 HCU Network America & OAA
Family Conference on June 26-27 in Bethesda, MD. This event will provide an opportunity to come together and share support when managing these rare metabolic disorders. More.
October is HCU Awareness Month! Homocystinuria is a group of rare inherited metabolic disorders that affects 1 in 200,000. Learn how you can get involved and raise awareness. More.
Immune Deficiency Foundation
The Immune Deficiency Foundation is proud to share they have received a $2.97 million grant extension from the US Health Resources and Services Administration (HRSA) to fund screening and education programs for people with severe combined immunodeficiency (SCID) through the SCID Compass program. More.
International Pemphigus and Pemphigoid Foundation
Registration is now open for the 2020 Virtual International Pemphigus and Pemphigoid Foundation Patient Education Conference: Creating a Brighter Future Together! Join with other patients, caregivers, researchers, industry partners and leading bullous disease experts October 2-4 about research and trends, disease management and awareness. More.
Melanoma Research Foundation
The Melanoma Research Foundation invites you to attend the 9th Annual Eyes on a Cure: Patient
& Caregiver Symposium on November 20-22 in Houston, TX. This free symposium is the largest annual meeting of ocular melanoma (OM) patients, caregivers, researchers and physicians to learn and connect. More.
Neuromuscular Disease Foundation
Neuromuscular Disease Foundation is proud to announce that Dr. Nuria Carrillo, formerly of NIH, has joined NDF's staff as Chief Medical Officer. More.
Siegel Rare Neuroimmune Association (SRNA)
SRNA invited Dr. Michael Levy and Dr. Elias Sotirchos to discuss the ABCs of NMOSD in their podcast, “Understanding Clinical Trial Results for NMOSD Therapies.” The experts explain the steps of a clinical trial, the requirements of FDA-approval, the differences between types of studies and three new NMOSD drugs. More.
SLC6A1 Connect
SLC6A1 Connect shares how equine therapy is helping with a 2-year-old's rare neurological disease. More.
The FPIES Foundation
Join the FPIES Foundation for Global FPIES Day, an annual online event
on October 14 to recognize food protein-induced enterocolitis syndrome and empower those living with this diagnosis by providing tools, support and resources for every day. Learn how you can get involved with #Allies4FPIES! More.
The Guthy-Jackson Charitable Foundation
The Guthy-Jackson Charitable Foundation presents a series of therapeutic breakout sessions with key stakeholders so patients can learn about new FDA-approved treatments for NMSOD. More.
The Marfan Foundation (The VEDS Movement)
The VEDS Movement, a division of the Marfan Foundation, invites you to participate in vascular Ehlers-Danlos syndrome (VEDS) action month this October! Take a look at the VEDS Movement website for action Items each week of the month to help with medical education and awareness for VEDS. More.
The RYR-1 Foundation
The RYR-1 Foundation is excited to announce the publication, "Clinical Care Guidelines: What Patients & Families Need to Know About RYR-1-Related Diseases." This handbook is written in non-technical, easily understood language to empower patients and families with RYR-1-related diseases to improve their care and quality of life. More.
Vasculitis Foundation
The Vasculitis Foundation's Patient and Family Conferences are going virtual! Register today for updates on treatments, research and to meet other patients. Wednesday, October 21 will focus on newly diagnosed patients and families, Saturday, November 14 is for advanced patients and families and Saturday, December 5 is for caregivers. More.
VHL Alliance
All VHL patients, families, friends and medical professionals are welcome to join the virtual VHL Alliance Annual Family Weekend on October 24-25. More.
Medical professionals and researchers will virtually unite at the International VHL Medical/Research Symposium October 29-31. You’re invited to come hear the keynote address, Toward a Treatment for VHL Disease: HIF2 Inhibitors. More.