Rare Disease Report

NORD News for July - The Voice of the Community

JULY 13, 2016
Guest post by NORD

FDA Commissioner to Speak at NORD Summit

Robert Califf, MD, commissioner of the US Food and Drug Administration (FDA), will speak at NORD’s annual Rare Diseases and Orphan Products Breakthrough Summit in October. Dr. Califf will provide a keynote address on the first morning of the Summit. The two-day event will take place October 17th and 18th at the Hyatt Regency Crystal City in Arlington, VA.
Dr. Califf was named FDA commissioner in February 2016. As FDA’s top official, he oversees programs and policies that enable the agency to carry out its mission to protect and promote the public health. Previously, before joining FDA in 2015, Dr. Califf was a professor of medicine and vice chancellor for clinical and translational research at Duke University.
NORD’s Summit will feature numerous other FDA senior officials, including Janet Woodcock, MD, director of the Center for Drug Evaluation and Research, and Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research.

Other highlights of the NORD Summit will include a telemedicine demonstration by David Flannery, MD, medical director of the American College of Medical Genetics; a panel on gene editing, gene therapy and how genetic innovation may affect those with rare diseases; and sessions devoted to commercial challenges and breaking down barriers to access.
Planners have created additional opportunities in the program this year for networking and interest group discussion. The full agenda will be available soon. Information regarding exhibiting, sponsorship and registration is on the NORD website.

August 19 is Deadline to Submit Poster Abstracts for NORD Summit

Academic, industry, government and patient organization researchers interested in presenting their rare disease or orphan product research to a large and diverse audience are invited to submit poster abstracts for the NORD Rare Diseases and Orphan Products Breakthrough Summit. The deadline is August 19. Abstracts are welcome from both students and professionals.
“Life-Transforming Treatments” will be the theme for the 2016 poster sessions. Under this over-arching theme, researchers are invited to submit within any of four areas:
  • Innovative Research
  • Medical Education Advancement
  • Patient Community Building
  • Other Topics
Notification of selected abstracts will be made by August 31. The Summit provides an opportunity to share promising research with a large and diverse audience that includes all rare disease stakeholder groups. The call for poster abstracts may be downloaded from the NORD website. Submissions should be sent by email to Ashley.Worrell@cbinet.com.

NORD Publishes Physician Guide to Mitochondrial Myopathies

As part of its ongoing series to promote medical professional awareness of rare diseases, NORD has published The Physician Guide to Mitochondrial Myopathies. This free online resource was written by Michelangelo Mancuso, MD, PhD, of the Neurological Clinic, University Hospital of Pisa, Italy, and Michio Hirano, MD, of Columbia University Medical Center in New York. It was made possible by an educational grant from Stealth BioTherapeutics. NORD’s Physician Guides are intended to promote earlier diagnosis and optimal treatment for patients with rare diseases. They are written for physicians and other medical professionals by independent medical experts who, with NORD, are solely responsible for the content.

Register for Rally for Medical Research Hill Day

Join NORD and more than 300 partner organizations to show support for medical research at the annual Rally for Medical Research Capitol Hill Day in September. This event is organized to encourage federal funding for medical research to save lives and promote a better quality of life for millions of Americans. Information and registration are available on the NORD website.


Amyloidosis Support Groups Provide Resources for Patients, Families and HCPs
A booklet and video on the Amyloidosis Support Groups website provides information for patients, their families and caregivers on the various types of amyloidosis. Included are listings of the major US and international treatment centers, as well as other resources.

Myocarditis Foundation Plans Family Support/Researcher Meeting
The Myocarditis Foundation’s 4th annual Family Support-Researcher Meeting will take place September 16-17 in Orlando. The event will draw together affected families, medical experts and researchers.

Pitt Hopkins Research Foundation Plans Scientific Symposium and Family Conference
The 2016 Pitt Hopkins Research Foundation Scientific Symposium and Family Conference will take place November 3-5 in Dallas.

NBIA Disorders Association to Celebrate 20th Anniversary
The NBIA (Neurodegeneration with Brain Iron Accumulation) Disorders Association will host an event in the Cincinnati area August 6-8 to observe the organization’s 20th anniversary and plan future research directions.

Myotonic Dystrophy Foundation Conference Set for September
The Myotonic Dystrophy Foundation will host its annual conference on September 15-17 in Washington DC. This event will include a patient-focused drug development meeting hosted by the Foundation to help educate federal agency officials about symptoms and disease issues that are important to patients and their families.

Tourette Syndrome Association Establishes Behavior Therapy Institute
Recognizing the critical shortage of trained behavioral therapists for treating Tourette syndrome and other tic disorders, the Tourette Syndrome Association of America Behavioral Science Consortium has developed the Tourette Syndrome Behavior Therapy Institute. The program is open to qualified and licensed healthcare providers including physicians, psychologists, social workers, nurses, occupational therapists and speech pathologists.

Vasculitis Foundation Regional Patient-Family Conference to Take Place in Kansas
The 2016 Vasculitis Patient –Family Conference will take place August 6 at the University of Kansas Edwards Campus in Overland Park, KS. This event will feature medical experts providing the most current information on treatments and research.

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