Rare Disease Report

NORD News for September - The Voice of the Community

SEPTEMBER 18, 2016
Guest post by NORD
News from NORD and its Member Organizations

The Hill Publishes Op-Ed by NORD CEO
Peter L. Saltonstall Urges Congress to Act Now to Help 15 Million Sick Children

The Hill, considered by many to be the premier source for policy and political news in the U.S., has published an op-ed piece by NORD President and CEO Peter L. Saltonstall urging Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher Program. The program encourages the development of therapies for children with rare diseases.
This program, which has been very successful, will expire if it is not reauthorized by September 30. Anyone wishing to join NORD in advocating for reauthorization of the program may do so here.
More than half of the 30 million Americans with rare diseases are children. Most have rare diseases for which there is currently no approved therapy. Rare diseases tend to be scientifically and medically complex. Patient populations are small and dispersed, making clinical trials difficult. The challenges are even greater when the patients are children.
Recognizing the need for more treatments for children with rare diseases, Congress in 2012 created the Rare Pediatric Disease Priority Review Voucher Program, which has resulted in six new therapies for seven rare diseases that previously had no treatment. The program awards priority review vouchers to companies that develop treatments for rare pediatric diseases. These vouchers can be used for a subsequent product or sold to another company. They allow companies to move through the FDA review process more quickly and, therefore, have great value to pharmaceutical or biotechnology companies.  
The program must be reauthorized by Sept. 30 or it will end, and the millions of children who have rare diseases with no treatment will lose a valuable ally in advancing the development of therapies.  

Deadline Extended to October 21 for NORD Research Grant Letters of Intent

The abstract submission deadline has been extended to Oct. 21 for NORD’s RFPs for grants to study the following diseases:
  • Alveolar capillary dysplasia with misalignment of the pulmonary veins
  • Appendix cancer and pseudomyxoma peritonei
  • Autoimmune polyglandular syndrome type 1
  • Homocystinuria
  • Malonic aciduria
  • Stiff person syndrome
Information about these grants and funding opportunities being provided by several NORD member organizations for the study of other rare diseases may be found on the NORD website.

Telemedicine, Gene Editing, Gene Therapy, and Transition to Adulthood for Pediatric Patients to be Among Topics at NORD Summit

Genetic innovation in diagnosis and treatment, and regulatory considerations for gene therapy, will be among the topics at the NORD Rare Diseases and Orphan Products Breakthrough Summit on October 17-18 in Arlington, VA. The program will include a session on Strategies to Address Patient Challenges, including emergency room visits and transition to adulthood. FDA will have a strong presence on several panels and with its own track. Breaking down barriers to access also will be a pervasive theme.
A new feature this year will be Lunch and Learn Breakout Roundtables, where discussion will focus on topics such as “Optimizing Communications among Patient Organizations and Industry”, “Partnering with Academia to Create Centers of Excellence”, and “Navigating and Accessing Hubs for Rare Disease Patients.”

Running For Rare Team to Run Oct. 8th Half-Marathon to Raise Funds for  Undiagnosed Patients

The Running for Rare Team is recruiting both runners and patient partners to help promote awareness of rare diseases and raise funds to assist undiagnosed patients. The team will run in the Eversource Hartford (CT) Marathon to take place on October 8 and other upcoming marathons and half-marathons.  
Funds raised by the team support the NORD/Undiagnosed Diseases Network (UDN) Patient Assistance Program. This program provides financial assistance to cover the cost of diagnostic testing for applicants to the Undiagnosed Diseases Network. Anyone wishing to make a donation to support the team’s efforts may write to runningteam@rarediseases.org.

Penn State Graduate Student Conducts Survey with NORD

Kerri Nelson, a graduate student at The Pennsylvania State University and mother of a child with a rare disorder, has designed a rare disorder toolkit to be disseminated to patients and families at an academic medical center. The purpose is to promote information and awareness of NORD’s website as a resource.
“When I began my Doctorate of Nursing Practice, I found the National Organization for Rare Disorders and felt that this organization would have been beneficial two years prior when my son was diagnosed with a rare disorder,” she wrote on the NORD blog. As a result, Nelson got the idea to help those with newly diagnosed children find and benefit from the NORD website. She has also developed a survey to learn how visitors to the NORD website found the site and what types of information they are seeking.


Pancreatic Action Network
The Pancreatic Action Network is accepting applications for research grants. Letters of intent are due Oct. 4.

Cholangiocarcinoma Foundation
The Cholangiocarcinoma Foundation invites investigators to submit research proposals for basic, translational, and clinical study of cholangiocarcinoma. The Foundation also has announced its 4th Annual International Conference to be held Feb. 1-3 in Salt Lake City.

Multiple System Atrophy Coalition
Multiple System Atrophy Coalition invites MSA patients, along with their families and care partners, and any interested healthcare professionals to attend the annual MSA Patient and Family Conference in New Orleans on Oct. 14-15.

American Brain Tumor Association
The ABTA has announced research grant funding opportunities.

Tuberous Sclerosis Alliance
The TS Alliance has organized its largest national event, with locations in 30 states. Register for a location near you.

The Global Annual CMTC-OVM Conference will take place in The Netherlands Nov. 11-12.

A-T Children’s Project
Nathaniel Heintz, PhD, and Michael Kastan, MD, PhD, longtime scientific advisers to the Ataxia-Telangiectasia Children’s Project, have been elected to the prestigious national academy of sciences.

Cutaneous Lymphoma Foundation
A patient conference is being scheduled for Oct. 29-30 in conjunction with the 3rd World Congress on Cutaneous Lymphomas at Columbia University in New York.

Narcolepsy Network
The 2016 Narcolepsy Network Conference will take place Oct. 21-23 at Lake Buena Vista, FL.

National Organization for Albinism and Hypopigmentation (NOAH)
A new and comprehensive resource has been developed to help parents and teachers support students with albinism.

Pachyonychia Congenita Project
The Pachyonychia Congenita Project has appointed a new executive director, Cindy Byers Altha.

Castleman Disease Collaborative Network
The second annual Quest for a Cure gala will take place on Nov. 4 in Philadelphia. The third annual Parents and Loved Ones Summit will take place Nov. 4-5 at the same location.

National Tay-Sachs and Allied Diseases Association
The 2016 Imagine and Believe Benefit will take place Nov. 10 in Cambridge, MA.

NephCure Kidney International
The Nephrotic Syndrome Regional Symposium Series has been announced with locations in Seattle, New York, Philadelphia and Miami.

National Spasmodic Dysphonia Association
A National Spasmodic Dysphonia Educational Conference will take place in Scottsdale, AZ, on Oct. 16.  

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