NORD News for December - The Voice of the Community

Guest post by NORD

News from NORD and its Member Organizations – December 2016

NORD Launches 2016 State Report Card and New Rare Action Network Website

A majority of states are not measuring up on legislative solutions to reduce the burden of living with a rare disease, according to a new report from the National Organization for Rare Disorders (NORD) Rare Action Network (RAN). The report, “State Report Card: A Roadmap to Improve the Lives of People with Rare Diseases,” indicates that progress is slow in several areas of health policy.

Those areas include, for example, coverage of medical foods, newborn screening and prescription drug cost-sharing limits. The new report is the second annual edition of the NORD RAN State Report Card. It expands upon the policy areas addressed in the previous report. A color-coded grading system is used to identify how well each state is doing.

“The ultimate purpose is to educate and empower advocates to affect change in their states,” said NORD Associate Director of State Policy, Tim Boyd. “We aim to provide actionable steps for states that will make meaningful improvements in peoples’ lives.”

The report was released on the same day NORD launched a new Rare Action Network website.  RAN is a network of advocates working together with NORD to encourage policies within states that address needs of rare disease patients and families. The website offers tools, training and resources to help individuals become effective advocates.

For Rare Disease Day in February and throughout the year, RAN and the new RAN website will offer a way for advocates to engage with NORD and others in their state, learn about issues and upcoming events, and track current topics and legislation.

21st Century Cures Act Reflects Years of Advocacy

NORD President and CEO Peter L. Saltonstall said that the 21st Century Cures Act signed by President Obama on December 13, for which NORD and other patient organizations have provided extensive advocacy, offers new hope to millions of Americans affected by rare diseases. Read his statement.   

Medical Foods Coverage for Military Families Would Be Expanded Under Bill Passed by Senate

The U.S. Senate on December 8 approved the National Defense Authorization Act (S. 2943) with language substantially expanding coverage for medical foods in the TRICARE program for military families. Medical foods are medically necessary nutrition essential for many people with rare diseases to survive.

NORD Can Provide Speakers and Resources for Rare Disease Day Events at Schools, Universities and Hospitals

Rare Disease Day will be observed on February 28, and NORD encourages medical professionals and students preparing for health-related careers to join others around the world in observing this day for awareness and education. At the national website for Rare Disease Day, NORD provides fact sheets and a toolkit specifically for those organizing Rare Disease Day events in schools or hospitals. To inquire about patient/caregiver speakers for Rare Disease Day events, write to

Nominations are Open for NORD’s Rare Impact Awards

January 13th is the deadline for submitting nominations for 2017 NORD Rare Impact Awards. These awards honor individuals and organizations for notable work in rare disease advocacy, science, patient care, ethics, research and public policy. The 2017 Rare Impact Awards celebration will take place in the amphitheater of the Ronald Reagan Building and International Trade Center, the largest structure in Washington DC. 


National Tay-Sachs & Allied Diseases Association Seeks Proposals
NTSAD is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies in the following diseases: Tay-Sachs, GM-1, Sandhoff  Canavan. Pre-applications are due January 4. Download 2016-2017 guidelines and other information. 

Association for Frontotemporal Degeneration Conference is Planned
The 2017 AFTD Education Conference will take place on May 5 at the Sheraton Inner Harbor Hotel in Baltimore. Online registration will open January 9. Read about the conference here.

Abstract Submission is Open for HHT International Scientific Conference
Authors are invited to submit abstracts for consideration for the 12th HHT International Scientific Conference for oral or poster presentations. Abstracts should include only original data and case reports must be submitted for poster presentation. The deadline is February 28.

International Waldenstrom’s Macroglobulinemia Foundation to Partner on Treatment Advances Workshop
The IWMF will partner with CancerCare on a Connect Education Workshop on “Advances in the Treatment of Waldenstrom’s Macroglobulinemia” to take place on January 11 from 12:30 to 1:45 pm ET. Featured speakers will be Stephen Ansell MD of Mayo Clinic and Jorge Castillo MD of Dana Farber. Participants can listen by phone or via Internet live-streaming. Advance registration is required, either online or by calling 800 813-4673. The workshop will be recorded and available later on the CancerCare website.

Hereditary Neuropathy Foundation Announces Newest Center of Excellence
Hackensack University Medical Center has been designated a center of excellence by the Hereditary Neuropathy Foundation. The designation was made as a result of the medical center’s clinical care, community engagement, research and education related to Charcot-Marie-Tooth disease/hereditary neuropathies.

Morgan Leary Vaughan Fund Provides Audio Podcast Series on NEC
The Morgan Leary Vaughan Fund has developed a new brochure to accompany its “Speaking of NEC: Necrotizing Enterocolitis” audio podcast series. The brochure provides a short introduction to each podcast. Copies have been distributed to 102 Neonatal Intensive Care Units in the U.S. 
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