NORD: The Voice of the Community - January 2018


NORD's Voice of the Community - January 2018

It’s been 35 years since dedicated patient advocates played a key role in enactment of the Orphan Drug Act and established NORD as the voice of the rare disease community. Throughout 2018, NORD will feature the historic accomplishments of those advocates at events such as the Rare Impact Awards in May and Rare Summit in October. The rare disease community has a proud history of advocacy, education, patient/family services and research. Watch for highlights throughout this anniversary year!

Join the 7,000 Mile Rare Movement

The 7,000 Mile Rare Movement is a new virtual campaign through which everyone can show their support for rare disease patients and caregivers. It is hosted by NORD as part of Rare Disease Day. Anyone can join. The goal is for participants to walk, run or cycle 7,000 miles during the month of February and to invite their families and friends to pledge a donation for every mile. Funds raised will support NORD’s programs and services for patients and their families. Participants can choose which specific program areas they would like to support. Learn more and sign up here.

NORD Issues Statement in Response to Senator Hatch’s Announced Retirement

Senator Orrin Hatch, one of the original sponsors of the Orphan Drug Act and other key legislation over the years, has announced that he will retire from the U.S. Congress at the end of 2018. NORD issued a statement of gratitude for his support for those living with rare diseases. “Over his 40 years in the United States Senate,” the statement says, “Senator Orrin Hatch has exhibited incredible dedication, leadership, and compassion for the rare disease community. We will miss him greatly.”

FDA Issues New Guidances On Orphan Drugs and Pediatric Diseases

Public comment is welcomed on an FDA draft guidance intended to improve the efficiency of drug development for rare pediatric diseases. Working with its counterpart in Europe, the European Medicines Agency (EMA), FDA has drafted an approach to pediatric rare disease drug development that could eliminate the need for certain clinical studies and reduce the number of patients who would receive a placebo rather than a potentially helpful drug. Read the draft guidance. The intent is to develop new, creative approaches to development of treatments for children with rare diseases — an area of significant unmet need – using Gaucher disease as a model. Learn more about this.

Public comment is also welcome on FDA’s plans regarding orphan drug designation for pediatric subpopulations of common diseases. More on this.

NIH Undiagnosed Diseases Network to Launch Grand Rounds Webinar Series

The NIH UDN will begin a webinar series on March 8 from 1 to 2 pm ET that will be free and open to advocates, students and others. The activity has been approved for free AMA PRA Category 1 credit. Each presentation will feature case studies of one or more undiagnosed patients. Participants will be able to ask questions and offer insights. Registration is required.

NORD Call for Abstracts Reopened

NORD has reopened the call to submit abstracts and letters of intent for research grants for study of the following rare conditions:

  • Cat eye syndrome
  • Malonic aciduria
  • Post-orgasmic illness syndrome

Additional information and the RFPs may be found on the NORD website. All U.S. and international researchers interested in studying these conditions are encouraged to apply.

Nominate Patients, Caregivers, or Advocates for FDA Patient Engagement Collaborative

Nominations are being accepted for the new FDA Patient Engagement Collaborative to improve and increase patient involvement in FDA matters. Nominations will be accepted through June 29, and those received after that date will be retained for possible future consideration. Read the Federal Register notice about this.


Several NORD Members to Speak at NIH 2018 Rare Disease Day Event

NORD board chair Marshall Summar MD will moderate a panel on which representatives of several NORD member organizations will speak at the 2018 Rare Disease Day event at the National Institutes of Health (NIH) on March 1. The event will take place on the NIH campus in Bethesday MD and is free and open to all. It will also be webcast. Read about the program and register to participate in-person or by webcast on the NIH website.

Alport Syndrome Foundation

2018 letters of intent for research funding are being accepted now by the Alport Syndrome Foundation, Pedersen Family, and Kidney Foundation of Canada, which are collaborating on research funding toward novel treatments to prevent kidney failure and hearing loss in patients with Alport syndrome. Learn more about this on the foundation’s website.

American Partnership for Eosinophilic Disorders

APFED is accepting applications for 2018 HOPE pilot grants. The program allows investigators from various disciplines to initiate studies relevant to eosinophil-associated diseases. Learn more on the APFED website.

Aplastic Anemia & MDS International Foundation

The AAMDSIF will be hosting seven “Living With Aplastic Anemia, MDS, PNH” patient and family conferences in cities throughout the U.S. Each free event offers learning opportunities from leading medical experts, connection with other patients and caregivers, and hope for the future. Visit the AAMDSIF website for additional information.

Hydrocephalus Association

The Hydrocephalus Association is soliciting applications for research o postinfectious and posthemorrhagic hydrocephalus. The goal is to provide seed funding for bold and innovative research with the potential to promote better understanding of disease mechanisms and development of novel therapies. Additional information is available on the association’s website.

Lipedema Foundation

The Lipedema Foundation seeks to hire a program associate to provide the foundation’s executive director with registry, database, and operations support. Applications are being accepted through the foundation’s website.

Lymphangiomatosis and Gorham’s Disease Alliance

LGDA has published funding opportunities on its website for study of lymphangiomatosis and Gorham’s disease. The period of award and level of funding are unique to each project.

National Niemann-Pick Disease Foundation

The board of the NNPDF is conducting a search for an executive director for the foundation. Founded 25 years ago, the NNPDF supports patients and families affected by Niemann-Pick disease, which affects fewer than 1,000 patients worldwide. Additional information is available on the foundation’s website.

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