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NORD News for July - The Voice of the Community

JULY 21, 2017

NORD Announces Keynote Speakers for its Breakthrough Summit

Food and Drug Administration (FDA) Commissioner Scott Gottlieb MD will present the keynote address in the opening session of the 2017 NORD Rare Diseases & Orphan Products Breakthrough Summit on Monday, October 16, in Washington DC. Award-winning journalist and founder/CEO of The Mighty Mike Porath will present the patient keynote.

Dr. Gottlieb, who was sworn in as FDA Commissioner in May 2017, previously served as FDA’s Deputy Commissioner for Medical and Scientific Affairs. Before that, he was Senior Adviser to the FDA Commissioner.

Mike Porath is founder and CEO of The Mighty. He has held writing, editing, and executive positions at ABC News, NBC News, The New York Times and AOL. He also is the father of a child with a rare syndrome known as Dup15q and sits on the board of Dup15q Alliance, a non-profit organization.

Register by August 25 for Early-Bird Pricing

Online registration is now open for the NORD Summit. Register by August 25 to save up to $400. The NORD Summit will take place October 16-17 at the Marriott Wardman Park Hotel in Washington DC.  

Submit a Poster Abstract or Become an Exhibitor or Sponsor

August 18 is the deadline to submit abstracts for the Summit Poster Session. The overall poster theme is “Life-Transforming Treatments”. In addition, there are opportunities to exhibit at the Summit or become a sponsor. Exhibiting and sponsorship inquiries may be sent to amoore@rarediseases.org.  

The NORD Summit is open to all and typically attracts more than 600 participants including leaders of patient organizations, NIH and FDA senior staff, those developing orphan products and ancillary educational, communications or industry partners.

Focusing on Community-Wide Topics

With a mix of plenary and breakout sessions, the Summit takes a community-wide approach to exploring how to advance research, innovative product development, patient access to care and patient/family quality of life. Topics will include:

  • Ethical guidelines for patient organizations and industry to interact
  • The future outlook for Patient Assistance Programs
  • Promoting earlier diagnosis
  • Next-generation treatments and advancing clinical trials
  • The challenge of healthcare costs and treatment prices
  • FDA perspectives on gene therapy and
  • A look at orphan approvals: an FDA perspective

 
Join NORD’s “Days of Action” Campaign to Protect Healthcare Coverage

Paul Melmeyer, NORD’s Director of Federal Policy, issued an update on the current status of healthcare reform on July 20th and urged members of the rare disease community to contact their members of Congress. The Senate leadership could move forward with a vote to repeal the Affordable Care Act very soon, he said, and both options currently being considered pose problems for those with rare diseases. Read Paul’s statement and learn how you can get involved. View a related video.
 
Sign Up to Receive Real-Time Policy Alerts From NORD

In this time of rapidly changing developments related to health policy, it’s important for all stakeholders to stay informed. NORD’s policy team works hard every day to represent rare disease patients and their families on key issues at both the state and federal level. You can sign up to receive up-to-the-minute policy updates from NORD, as well as news about how you can add your voice to the conversation. You can also join the Rare Action Network to participate in advocacy within your state.
 
Examining the Role of Caregivers at NINR Caregiving Summit

NORD Vice President of Educational Initiatives Mary Dunkle will speak on a panel discussing the role and needs of caregivers at “The Science of Caregiving: Bringing Voices Together” to be hosted by the National Institute of Nursing Research on August 7-8 in Bethesda. This conference is open to all and will also be livestreamed and archived for on-demand viewing. 
 
Columbia Neurosurgery Article Highlights Medical Expert’s Support for NORD Patient Information

An article on the Columbia University Medical Center website describes how neurosurgeon Jeffrey Bruce, MD, helped NORD develop a report for patient/family education on anaplastic astrocytoma. Dr. Bruce is one of many rare disease medical experts who volunteer their time and expertise to help NORD provide information on rare conditions for patients and their families.
 
NEWS FROM NORD MEMBER ORGANIZATIONS

NORD Welcomes All Things Kabuki as New Member

All Things Kabuki, an organization providing awareness, education and support on behalf of those affected by Kabuki syndrome, is NORD’s newest member organization. Read NORD’s Rare Disease Database report on Kabuki syndrome.

Alpha-1 Foundation Announces Funding Opportunities
The Alpha-1 Foundation has announced its funding opportunities in its 2017-2018 grants cycle. In-cycle Letters of Intent are due by September 22 and may be submitted online.

Aplastic Anemia & MDCS International Foundation Seeks Development Officer
AAMDS is seeking a Development Officer to join their small yet committed and passionate staff working to make a difference in the lives of patients and families affected by life-threatening bone marrow failure diseases. For more information, click here.  

cureCADASIL Announces Publication of Study Results
cureCADASIL Association has announced the publication of a study by Dr. Arboleda-Velasquez in the Journal of Experimental Medicine. CADASIL is a disorder of the small blood vessels that causes subcortical transient ischemic attacks or strokes and progresses to early vascular dementia.

Hyper IgM Foundation Publishes Blog
The Hyper IgM Foundation recently published a blog promoting better understanding of Hyper IgM syndrome, a rare, genetic, immunodeficiency disorder and, in particular, seeking to provide information for patients and families going through stem cell transplantation.

International Pemphigus & Pemphigoid Foundation Annual Conference is Planned
The 20th annual IPPF Patient/Family Conference will take place September 15-17 in Newport Beach, CA.  

MSA Coalition Plans Patient/Family Conference
The MSA (Multiple System Atrophy) Coalition Patient/Family Conference will take place October 13-14 in Nashville.

Neuroendocrine Tumor Research Foundation Issues Request for Proposals
NETRF has issued an invitation for Letters of Intent for 2017 research grants. Proposals are being sought with the potential to transform understanding of neuroendocrine tumors and/or lead to improved treatments and diagnostics for patients. Letters of Intent may focus on any type of NET and may be for basic, translational or clinical research.

Spinal CSF Leak Foundation and Cedars-Sinai Plan Intracranial Hypotension Symposium
A full-day multidisciplinary symposium on intracranial hypotension secondary to spinal cerebrospinal fluid (CSF) leak will bring together top clinicians and researchers to share the latest advances in diagnostics and treatments for this under-diagnosed but treatable secondary headache disorder. This event will take place Saturday, Oct. 14, in Santa Monica, CA.
 

 


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