Rare Disease Report

The Voice of the Community: News from NORD and its Member Organizations May 2017

MAY 16, 2017
Guest Post by NORD

NORD Launches Free CME Program to Educate Medical Professionals About Rare Diseases

On June 17th in Washington DC, NORD will host the first event in a new Continuing Medical Education (CME) program to educate medical professionals about rare diseases. Additional information and online registration are available here.

The program, provided jointly with the University of Massachusetts Medical School, is designed for general practitioners, emergency room physicians and staff, pediatricians, and family medicine practitioners and staff. The overall mission is to raise awareness of the prevalence of patients affected by rare diseases, promote earlier diagnosis, and improve patient outcomes.

The event on June 17th, titled Finding a Zebra Among Many Horses, will cover diagnostic hurdles and tools and resources for diagnosis. It will include demonstration of resources in areas such as rare disease advocacy, education, and research. 

Abstracts and Letters of Intent Being Accepted Now for NORD 2017 Research Grants

June 23rd is the deadline to submit abstracts and letters of intent for 2017 research grants through the NORD Research Program. These grants are made possible by donations to NORD, often from rare disease patients and their families. In some cases, the grants are co-sponsored by NORD and disease-specific patient organizations.
In the current funding cycle, grants are available for the study of five rare diseases:
  • Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV)
  • Appendix cancer and pseudomyxoma peritonei (PMP)
  • Cat eye syndrome
  • Malonic aciduria
  • Post-orgasmic illness syndrome
Information is also available on the NORD website about current research funding opportunities from NORD member organizations.

NORD and National Health Council Submit Sign-on Letter to Congress Urging Swift Reauthorization of FDA User Fees

NORD and the National Health Council have submitted a letter to members of Congress signed by more than 120 patient advocacy organizations urging swift reauthorization of FDA user fees. The letter states that the current FDA user fee agreements are the culmination of months of negotiation between FDA and the medical product industry, with significant input from the patient advocacy community, and are “critical funding mechanisms” for FDA programs.

NORD Says American Health Care Act Jeopardizes Health and Well-Being of Rare Disease Patients

After the U.S. House of Representatives passed the American Health Care Act (AHCA) recently, NORD issued a statement saying “We will continue to do everything we can to prevent this legislation as it is currently written from being enacted.”

NORD said the AHCA jeopardizes the health and wellbeing of rare diseases patients by allowing states to opt out of critical pre-existing conditions protections, as well as the prohibition on annual and lifetime caps and community rating.


Children’s Cardiomyopathy Foundation Announces Research Funding Availability

The Children’s Cardiomyopathy Foundation is offering one-year research grants for studies focused on pediatric cardiomyopathy. Letters of intent are due June 14.

Cutaneous Lymphoma Foundation Conference to Take Place June 24-25

The annual two-day patient conference of the Cutaneous Lymphoma Foundation will be held June 24-25 in the Los Angeles area. The event will feature clinical and non-clinical presentations.

DUP15q Alliance to Host Scientific Symposium and Family Conference

On July 27-28, the DUP15q Alliance will host its 2017 Science Symposium at the Luskin Conference Center, UCLA. On July 24-26, the 9th International DUP15q Alliance Family Conference will take place at Redondo Beach, CA.

KIF1A.org Features Interview with Wendy Chung, MD

KIF1A.org, which has recently joined the NORD community of members, is featuring a video interview with one of their board members, Wendy Chung, MD, PhD, on their website to promote awareness of the urgent need for research on KIF1A, a rare, genetic syndrome. Dr. Chung directs the clinical genetics program at Columbia University Medical Center and conducts research on a wide range of human genetic disorders.

Myotonic Dystrophy Foundation and Wyck Foundation Offer Training Fellowships

September 1 is the deadline for applications for training fellowships being offered by the Wyck Foundation and Myotonic Dystrophy Foundation.

Neuroendocrine Tumor Research Foundation Grants Announced

The 2017 Neuroendocrine Tumor Research Foundation grants have been announced, infusing $4 million of new funding into neuroendocrine tumor research to drive development and application of precision therapies. This is the largest commitment NETRF has ever made in a single year and was made possible by the Margie and Robert E. Petersen Foundation.

Oley Foundation Hosts Webinar Series

Upcoming presentations in the Oley Foundation webinar series include a program on May 30 on “Traveling with IV Nutrition or Tube Feeding” and one on June 20 on “Treatment Options for Gastroparesis”.  

Organic Acidemia Association Launches Natural History Study with NORD

In collaboration with NORD, the Organic Acidemia Association has launched an international natural history study to support research on rare organic acid disorders, which cause multiple life-threatening conditions.

PSC Partners Seeking a Cure to Host Patient Conference

On June 23-25, PSC (Primary Sclerosing Cholangitis) Partners Seeking a Cure will host a patient conference in Cleveland, Ohio, in partnership with the Cleveland Clinic Transplant Center and Digestive Disease and Surgery Institute.

Pulmonary Hypertension Association and ATS to Host PH Program

On May 20 in Washington DC, the Pulmonary Hypertension Association and the American Thoracic Society will host a program in which participants can learn from leading PH experts and meet other patients, families and caregivers. The event is part of a Meet the Experts session hosted by PHA and the American Thoracic Society’s Public Advisory Roundtable.

Registration is Open for Spinal CSF Leak Foundation Symposium

The Spinal CSF Leak Foundation will host an Intracranial Hypotension Symposium on October 14 in Santa Monica, CA, in partnership with Cedars-Sinai. Registration is now open.

Tuberous Sclerosis Alliance Sponsors Patient-Focused Drug Development Meeting With FDA

June 21 is the date of a Patient-Focused Drug Development meeting sponsored by the Tuberous Sclerosis Alliance with the Food and Drug Administration at the Hyatt Regency on Capitol Hill in Washington, DC from 8:30 a.m. to 4:30 p.m. The purpose of the meeting is for individuals affected by TSC and caregivers to communicate their perspectives on living with TSC to FDA staff members. The meeting is free, open to the public and will be webcast. The morning session will focus on parents of children with TSC who have experienced, or are at risk for developing, epilepsy. The afternoon will focus on adults with TSC and/or LAM who have experienced or are at risk for developing angiomyolipomas or LAM. Online registration is available.

Vasculitis Foundation Plans International Symposium

The Vasculitis Foundation’s 2017 International Vasculitis Symposium will take place June 23-25 in Chicago. Hundreds of autoimmune vasculitis patients will join dozesn of medical specialists to learn about the latest advances in vasculitis research, disease therapies, and clinical studies. 

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