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NORD News for March - The Voice of the Community

Published Online: Monday, Mar 20, 2017

NORD Announces Honorees for 2017 Rare Impact Awards

NORD hasHR1313 announced the individuals and organizations who will be honored for their accomplishments on behalf of the rare disease community at the annual NORD Rare Impact Awards celebration in Washington DC on May 18th.

Each year, Rare Impact Awards are presented to:
  • Patients, caregivers and patient advocates
  • Clinicians and/or medical researchers
  • A NORD member organization
  • Industry innovators for diagnostic or treatment advances
 
The Rare Impact Awards is a charity event celebrating those who are leading efforts to make a difference for the millions of Americans living with rare diseases and a benefit to raise funds for NORD’s programs and services on behalf of all affected individuals and families.
 
The 2017 event will feature nationally touring music artist and winner of NPR Music’s 2016 Tiny Desk Contest, Gaelynn Lea, who will perform original songs, and the David Bach Consort. The event will take place in the Ronald Reagan Building in Washington DC.
 
To purchase tickets to the 2017 Rare Impact Awards celebration or read about advertising, sponsorship and donation opportunities, visit the website.

NORD Awards Seven Rare Disease Research Grants

NORD has awarded seven new grants to support research on rare diseases. These grants were awarded to researchers who responded to NORD’s 2016 call for proposals. Requests for proposals for 2017 will be published on the NORD website soon.
 
The new grants provide funding for study of the following diseases:
  • Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins (with support funding raised by The David Ashwell Foundation and the Alveolar Capillary Dysplasia Foundation)
  • Appendix Cancer and Pseudomyxoma Peritonei (PMP) (with support funding raised by The Appendix Cancer Pseudomyxoma Peritonei Research Foundation)
  • Autoimmune Polyglandular Syndrome Type 1 (with support funding raised by the APS Type 1 Foundation)
  • Homocystinuria due to Cystathionine Beta-Synthase Deficiency (with support funding raised by public donations)
  • Malonic Aciduria (with support funding raised by The Hope Fund, Lundbeck’s “Raise Your Hand” campaign, and public donations)
  • Stiff Person Syndrome (with support funding raised by Lundbeck’s “Raise Your Hand” campaign)
 
Recipients of the 2016 NORD Research Grants are:
  •  
  • Przemyslaw Szafranski, PhD, Baylor College of Medicine
  • Venkatesh Govindarajan, PhD, Creighton University
  • David L. Morris, MD, PhD, St. George Hospital, Australia
  • Maureen A. Su, MD, University of North Carolina at Chapel Hill
  • Warren D. Kruger, PhD, The Research Institute of Fox Chase Cancer Center
  • Michael J. Wolfgang, PhD, Johns Hopkins University School of Medicine
  • Sarah Crisp, MB, Bchir, PhD, University College London
 

NORD Issues Statement Opposing Preserving Employee Wellness Programs Act (H.R. 1313)

Pledging to “do everything in our power to prevent this bill from moving forward in its current form,” NORD has announced its opposition to H.R. 1313, which exempts employee wellness programs from critical patient protections included in the Americans with Disabilities Act (ADA) and Genetic Information Nondiscrimination Act (GINA).

AHCA Maintains Key Patient Protections But May Present Access Challenges, NORD Statement Says

After House Republicans released their replacement package for the Affordable Care Act, to be known as the American Health Care Act, NORD released a statement in which it compared provisions in the AHCA with the Principles for Health Coverage Reform that NORD put forward recently on behalf of its 260 member organizations. 

“After comparing the AHCA to our principles,” the statement notes, “we find that several key patient protections are maintained, but changes to the private insurance market and Medicaid program may jeopardize access to affordable, quality health care for rare disease patients. We look forward to working together with Congress to resolve these potential patient access issues.”

Pamela Gavin of NORD to Speak at Technology and Rare Neurological Diseases Symposium

NORD Chief Operating Officer Pamela Gavin will speak at a symposium to be hosted by the University of Rochester on May 12 on “Technology and Rare Neurological Diseases.” Registration is open for this event, which will focus on novel applications of technology to accelerate the development of new therapeutics for individuals with rare neurological disorders.

FDA To Host Public Workshop on Engaging with CDER

A public workshop, “Roadmap for Engaging with FDA’s Center for Drug Evaluation and Research”, will take place on Friday, May 12, from 9 am to 3 pm at FDA’s White Oak Campus in Silver Spring, MD. Registration is free and based upon space availability. For information, contact Chris Melton at NAV-CDER@fda.hhs.gov.
 

NEWS FROM NORD MEMBER ORGANIZATIONS

APS Type 1 Symposium is Planned
The second APS Type 1 Symposium for physicians and families will take place July 13-15 at the State University of New York at Stony Brook. Additional information will be available from the APS Type 1 Foundation.

Children’s Cardiomyopathy Foundation to Host Webinar
CCF will host a webinar – “An Overview of Genetic Testing for Cardiomyopathy Families” – on March 29. Join Allison Cirino, genetic counselor at the Brigham and Women’s Hospital, for an overview of genetic testing including new developments in the field. Register online.

 
Foundation for Prader-Willi Research Highlights Study of Genetic Therapy for Prader-Willi Syndrome
The Foundation for Prader-Willi Research has published a blog that discusses a new study which shows promising first steps toward genetic therapy for Prader-Willi syndrome. 
 
HHT International Scientific Conference is Planned
Early bird pricing is in effect until April 15th for the 12th International HHT Scientific Conference, to take place June 8-11 in Croatia. Register online or contact Cure HHT for more information.
 
Myasthenia Gravis Foundation of America and New York Academy of Sciences to Host Conference
The 13th International Conference on Myasthenia Gravis and Related Disorders will take place May 15-17 at the New York Academy of Sciences, co-hosted by the Academy and the Myasthenia Gravis Foundation of America.
 
Myocarditis Foundation and Texas Children’s Hospital to Host Conference
A one-day seminar on pediatric myocarditis is planned for Saturday, March 25. It will be hosted by the Myocarditis Foundation, in conjunction with Texas Children’s Hospital, where the conference will take place.
 
Myotonic Dystrophy Foundation Promotes Involvement in Clinical Studies and Trials
The Myotonic Dystrophy Foundation is launching an “Every Body Counts!” campaign to encourage participation in clinical studies and trials. The Foundation will promote awareness of research and trial opportunities currently available to community members and share stories from study participants.
 
National Adrenal Diseases Foundation Publishes Report on Survey
NADF, with support from Opinion Health, conducted a national survey for healthcare professionals on adrenal insufficiency. Specifically, the purpose was to better understand current diagnosis and management of AI, particularly among patients with Addison’s disease. Now NADF has published a report on the survey findings.
 
NTM Info & Research Announces Patient Scholarships
Patient scholarships are now available for the 2017 NTM & Bronchiectasis Physician/Patient Conference to be held at Georgetown University in Washington DC on Friday, May 19. Scholarship applications are due April 15th.
 
Sarcoidosis Awareness Month to be Observed in April
The Foundation for Sarcoidosis Research will lead a month-long awareness campaign during April. The Foundation is also planning patient education conferences over the next several months in Washington DC, Seattle WA, Ann Arbor MI, and Jacksonville FL. Visit the website for additional information.
 
“Zumbathon” Planned to Raise Funds for SYNGAP Research
This family “Zumbathon” will take place on Saturday, April 29, in the Indianapolis area to raise funds for research supported by the Bridge the Gap – SYNGAP Research and Education Foundation. 
 


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