Rare Disease Report

NORD: The Voice of the Community - December 2017

DECEMBER 27, 2017
NORD Extends Holiday Greetings to All!
The NORD staff sends best wishes for peaceful, healthful holidays for all, and for 2018 to be a year of significant advances for all who live with rare diseases.

Sending Gratitude to the Entire Community for Joining Together to Support Orphan Drug Tax Credit
As the end of a challenging year approaches, NORD has sent a message of gratitude to the entire rare disease community for coming together to make our voices heard on the need to preserve the Orphan Drug Tax Credit (ODTC). While the tax reform legislation ultimately passed by Congress does reduce the ODTC, it doesn’t eliminate it entirely, which could have been the outcome.

NORD believes the many community members who took the time to contact their legislators and made their voices heard were extremely important on this issue. NORD’s Director of National Policy Paul Melmeyer has posted a video thanking all the rare disease advocates who supported this cause.

NORD Awards Five New Grants for Rare Disease Research
Research grants have been awarded to scientists at the following institutions for the study of rare diseases through the NORD Rare Disease Research Grant Program:
  • Cincinnati Children’s Hospital Medical Center
  • University of California, San Diego
  • Uniformed Services University of the Health Sciences in Bethesda
  • Institut National de la Sante et de la Recherche Medicale (INSERM)
  • University of South Carolina School of Medicine

NORD Publishes Three New Physician Guides and Disease Reports
As part of its Physician Guide series to promote earlier diagnosis and optimal treatment for people with rare diseases, NORD has published new guides for physicians on three topics: hepatocellular carcinoma (HCC), acute myeloid leukemia (AML) and pigmented villonodular synovitis (PVNS). In addition, NORD has published corresponding reports on those topics for patients and caregivers. The new resources were made possible by an educational grant from Daiichi Sankyo, and the content was developed by NORD and independent medical experts. 

January 12 is Deadline for Rare Impact Awards Nominations
Don’t forget to nominate a patient, caregiver, colleague or public official for a 2018 Rare Impact Aware. These awards honor individuals, organizations or companies that have had a positive impact in the fight against rare diseases.
They are presented each year in May at NORD’s annual gala celebration and fundraiser in Washington DC. The program will have special significance in 2018 because that year will mark the 35th anniversary of NORD and the Orphan Drug Act.
Awards will be given in four categories to match the four pillars of NORD’s mission:
  • Advocacy
  • Education
  • Patient Assistance
  • Research
Honorees have included patients, caregivers, healthcare professionals, researchers, legislators, and government agency staff. Nominations may be submitted through the NORD website.

NORD Publishes New Report for Patients on Li-Fraumeni Syndrome
A new report for patients and caregivers on Li-Fraumeni syndrome has been added to NORD’s Rare Disease Database. Reports in the database are available free to all around the world. The new report was developed in collaboration with Holly Fraumeni and Robert Lufkin DO of the Li-Fraumeni Syndrome Association, one of NORD’s member organizations.


NORD Welcomes Five New Member Organizations
The following organizations have been approved for membership by NORD’s Board of Directors: Advocacy & Awareness for Immune Disorders Association (AAIDA), Amniotic Fluid Embolism (AFE) Foundation, the Atypical HUS (aHUS) Foundation, HCU Network America, and the Lung Transplant Foundation (LTF).
Podcast Series on NMO is Available from Guthy-Jackson Charitable Foundation
A new podcast series, NMO and the Power of Rare, is now available to all and covers topics related to neuromyelitis optica. Episode 1 of the series is titled “The NMO Revolution: The Top 5 NMO Breakthroughs in the Past 5 Years.” It features Nancy Sicotte MD, director of the MS Program at Cedars-Sinai Medical Center. 

Alagille Syndrome Alliance Seeks a Development Officer
The Alagille Syndrome Alliance, based in Portland, OR, representing those affected by the rare genetic syndrome known as Alagille syndrome, has an opening for a Development Officer.

Cornelia de Lange Syndrome (CDLS) Foundation is Accepting Abstracts for Symposium
Abstracts are being accepted for the Eighth Biennial Scientific and Educational Symposium of the Cornelia de Lange Syndrome Foundation, to take place June 27-28 in Minneapolis. The meeting features talks by leading researchers and, in addition to invited speakers, several platform talks are chosen from submitted abstracts. The deadline to submit an abstract is Feb. 15. Send abstracts and/or questions to kimballcdls@gmail.com or call Amy Kimball at 443 849-3012.

January 15 is Deadline to Indicate Interest in Homocystinuria Research Grants
HCU Network America and HCU Network Australia have announced a joint call for expressions of interest for 2018 research grants. Additional information is available here.

Osteogenesis Imperfecta Foundation to Host Conference in July
The Osteogenesis Imperfecta Foundation will host its biennial national conference in Baltimore July 13-15. The program will feature a full lineup of sessions on medical and practical living topics, forums to connect with leading OI experts, research updates, and more.

Pre-Applications Due January 12 for Tay-Sachs Research Funding
NTSAD (National Tay-Sachs & Allied Diseases) and CTSF (Cure Tay-Sachs Foundation) are jointly soliciting proposals for research funding for study of Tay-Sachs and several related diseases.

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