NORD News for April - The Voice of the Community

Guest post by NORD

NORD to Host Rare Impact Awards on May 17th

Honorees have been announced for the 2016 Rare Impact Awards, to be hosted by NORD on May 17 at the Warner Theatre in Washington DC. This annual event, which is open to all, celebrates individual and organizational achievements to drive progress for those living with rare diseases.

The 2016 honorees are:





For those attending this event, NORD has reserved a hotel room block at the JW Marriott Hotel, which is near the theatre. Visit the NORD website to register for the event, read about sponsorship opportunities and link to hotel reservations.

NORD and Its Members Exhibit at Medical Student Convention

NORD hosted a “Meet the Patients” event in its exhibit booth at the annual convention of the American Medical Student Association (AMSA) in Arlington, VA, on April 1 -2. Several patients and caregivers shared their stories of rare disease diagnosis and treatment with the students who visited NORD’s booth.

Jana and Stephen Monaco, Organic Acidemia Association; Ike Casey, CDLS Foundation; Elaine Rose, Association of Frontotemporal Degeneration; Elizabeth Hoey, NORD; Lynn Lothian, Alpha-1 Foundation; Carol Hoxie, Platelet Disorder Support Association, and Mary Dunkle, NORD.

Laura Brockwell, CFC International; Kevin O'Brien, Alport Syndrome Foundation; Mary Dunkle, NORD; Stacy Hardy, Cushing's Support and Research Foundation; and Elizabeth Hoey, NORD.

Jana and Stephen Monaco, Organic Acidemia Association; Ike Casey, CDLS Foundation; Elaine Rose, Association of Frontotemporal Degeneration; Elizabeth Hoey, NORD; Lynn Lothian, Alpha-1 Foundation; Carol Hoxie, Platelet Disorder Support Association,;and Mary Dunkle, NORD.

NORD member organizations who had representatives in the booth were: Cornelia de Lange Syndrome Foundation; Alport Syndrome Foundation; Organic Acidemia Association; Platelet Disorder Support Association; CFC International; Cushing’s Support and Research Foundation; Alpha-1 Foundation; Association for Frontotemporal Degeneration; CMTC-OVM US; CurePSP; CureHHT; PRISMS; Jack McGovern Coats Disease Foundation; Pulmonary Hypertension Association; Narcolepsy Network; and International Pemphigus & Pemphigoid Foundation.

“It’s so much more interesting and memorable to meet the patients and parents first-hand rather than simply reading a few sentences about a rare disease in a textbook,” one student told the NORD representatives.  This was the second year that NORD has hosted an event of this type at the annual AMSA convention.

In NORD’s Rare Action Network™: Illinois Edges Closer to Establishing Rare Disease Commission

The Illinois House of Representatives this week passed HB 4576. This House Bill seeks to establish a Rare Disease Commission that would give patients, many of whom are too young or too sick to speak for themselves, a voice in the state government. The bill would also provide educational resources for elected officials on critical issues related to rare diseases and access to treatment.  The bill now moves to the Illinois State Senate. Those interested in joining NORD in advocacy for this bill should write to Tim Boyd at

Runners and Patient/Caregiver Partners Are Being Recruited for 2016 Running for Rare Team

The Running for Rare marathon team, which has raised more than $500,000 to help rare disease patients over the past nine years, is accepting applications from runners and “community partners”. The team began as a grassroots effort with four runners and, by 2015, had grown to more than 100 runners. Through NORD, each runner is paired with a patient or caregiver partner to provide inspiration and encouragement.

Both runners and partners describe being part of the team as a positive, life-changing experience. The team this year became officially affiliated with NORD, and all funds raised support NORD’s patient assistance program for patients applying to the NIH Undiagnosed Diseases Network.  


National Brain Tumor Society CEO Named to Cancer Moonshot Initiative’s Blue Ribbon Panel of Experts

David F. Arons, JD, CEO of the National Brain Tumor Society, has accepted an invitation to serve on the Blue Ribbon Panel of the National Cancer Advisory Board to help inform the scientific director of the National Cancer Moonshot Initiative announced by President Obama earlier this year.

FIRST National Conference to Take Place June 24-26 in San Diego

The Foundation for Ichthyosis & Related Skin Types (FIRST) will host its annual conference in San Diego on June 24-26. This conference offers a unique opportunity to learn about these conditions and meet the medical experts one-on-one.

May is Ichthyosis Awareness Month. Learn how you can get involved here.

CurePSP to Host Medical Professionals Education Day

CurePSP (progressive supranuclear palsy) and Johns Hopkins University will host a Medical Professionals Education Day on May 16 at Johns Hopkins Hospital. The purpose will be to enhance medical professionals awareness of atypical Parkinsonian disorders.  This interactive half-day workshop is free and open to all medical professionals.

CurePSP is also co-hosting a Patient and Medical Professionals Education Day: Focus on PSP with the University of Pennsylvania and Good Shepherd Penn Partners on June 2. 

Lieber Scientists Study Pitt-Hopkins Syndrome

A recent Baltimore Sun article describes work being done by scientists at the Lieber Institute for Brain Development to better understand a rare form of autism known as Pitt-Hopkins syndrome.

May is International Behcet’s Awareness Month

May 20th is Behcet’s Awareness Day and the entire month of May is Behcet’s Awareness Month. A Walk for Behcet’s is planned for May 22 in Rochester, MI.

American Brain Tumor Association Offers Free One-Day Educational & Networking Meetings

ABTA will host free one-day meetings in communities across the US for patients, family members and caregivers to gather up-to-date brain tumor information from leading experts, receive guidance on managing symptoms and network with each other. Upcoming meetings are planned in Denver, Atlanta and Arlington, VA.

First International SYNGAP1 Conference Scheduled

This conference will take place at Texas Children’s Hospital Nov. 30-Dec. 1. It will utilize a novel, multidisciplinary approach to bring together patients and families, clinicians and researchers as equal stakeholders to accelerate discovery.
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