NORD News for August 2017 - The Voice of the Community

NORD Summit to Feature Key Topics for the Entire Community

“Ethical Guidelines for Patient Organizations and Industry to Collaborate”, “The Challenge of Healthcare Costs and Treatment Prices”, “Promoting Earlier Diagnosis” and “The Power of Data-Sharing” will be among the topics at the NORD Rare Diseases and Orphan Products Breakthrough Summit October 16-17 in Washington DC.
The Summit is for the entire rare disease community, and an estimated 600 participants will attend, representing more than 80 patient organizations, FDA, NIH, and the pharmaceutical industry.
Early-bird pricing is in effect until August 25. Registration may be done online. August 18 is the deadline to submit poster abstracts.

Share Your Story; Do Your Share

Earlier this year, NORD released a video that was the start of a year-long campaign to raise national awareness of rare diseases and how to help. It’s easy to do your share for the rare disease community! #DoYourShare today by sharing a video on our new website. Tell us how you think people can do their share to improve the lives of people with rare diseases or share your own Do Your Share Moment. Submit a video.

Runners and Community Partners are Needed

In October, NORD’s signature charity running team, “Running for Rare”, will participate in the Eversource Hartford (CT) Full and Half Marathon to benefit the rare disease community. Each runner is paired with a community partner to provide inspiration and to promote awareness of specific rare diseases. Runners and community partners are needed for the Hartford event. Apply here.

Rare Disease Film Festival is Planned for October 3

This first-ever event will showcase films from around the world that address the challenges of living with a rare disease. It will take place October 3 in Boston. View the new trailer and learn more here.

Free Rare Action Network Meetings Are Being Held in the Carolinas This Fall

On September 13 and 14, the Rare Action Network – the nation’s largest rare disease advocacy network – will host free advocacy and networking meetings in the Carolinas. These meetings will provide opportunities to meet other rare disease advocates, connect with NORD’s RAN team and learn how to strengthen your voice as an advocate.   


AA MDS Offers Webinar Series
The AA MDS International Foundation is accepting registration for three webinars to be offered in August. They are: “MDS Non-Transplant Treatments: What’s in the Pipeline?” on Thursday, Aug. 17; “PNH Clonal Expansion and Prognosis to MDS” on Thursday, Aug. 24; and “MDS Research Updates from ASCO and EHA in 2017” on Tuesday, Aug. 29.
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.
CMTC-OVM to Celebrate 20th Anniversary
CMTC-OVM (Cutis Marmarata Telangiectasia Congenita) will host its annual member conference and celebrate its 20th anniversary on Saturday, Nov. 4, at the Hotel Leusden in The Netherlands. Additional information.
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.
Immune Deficiency Foundation Names New President and CEO
John G. Boyle has been named president and CEO of the Immune Deficiency Foundation, succeeding IDF President and Founder Marcia Boyle, who has retired.
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.
Pulmonary Hypertension Association Offers “PHA On The Road”
PHA on the Road is a FREE day-long education and networking program that may be coming to a city near you. This patient-focused program offers interactive presentations, educational sessions, opportunities for patients and their families to meet others living with PH and speak with local PH experts. #PHAontheRoad has two more cities remaining on the schedule for this year: Portland OR and Pittsburgh PA. Don’t miss the chance to meet others living with PH, caregivers and local PH specialists in your region. Register today.
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.
Vestibular Disorders Association Celebrates Balance Awareness Week
Vestibular (inner ear and brain) disorders affect patients physically, mentally and emotionally, causing chronic dizziness, vertigo, imbalance, tinnitus, depression and anxiety. To promote awareness of these invisible conditions, the Vestibular Disorders Association celebrates Balance Awareness Week on September 18-24. Visit the website for more information. 
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