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Rare Diseases Constitute a Public Health Issue NCATS Director

Rare Diseases Constitute a Public Health Issue NCATS Director

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Rolfs Arndt, CEO of German genetics diagnostic testing company Centogene, speaks at WODC USA.

... Rare Disease Research. Gaylord Entrance to Gaylord National Convention Center in Oxon Hill, Maryland, site of WODC USA.

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease

Leonard Lance speaks at NIH in Bethesda, Maryland. (Photo by Larry Luxner)

Anne Pariser directs the NCATS Office of Rare Diseases Research in Bethesda, Maryland. (Photo courtesy NCATS)

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children's National

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

Marshall Summar, director of the Rare Disease Institute at Children's National, will speak at WODC 2019. (Photos by Larry Luxner)

Pin It on Pinterest. Huntington's Disease News. Rare Diseases Constitute a 'Public Health Issue,' NCATS Director Warns

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Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

Pin It on Pinterest. SMA News Today. Rare Diseases Constitute a 'Public Health Issue,' NCATS Director Warns

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

Hunter Pageau, 12, speaking at a Rare Disease Day event in Connecticut. He has a rare form of SMA called SMARD. (Photo by Larry Luxner)

ICD-10 Codes, 'Really Important' to Rare Disease Patients, Soon Up

Rare Disease Groups Welcome FDA's Embrace of 'Real World' Data in Clinical Trials

Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

Pin It on Pinterest. Muscular Dystrophy News · Rare Diseases Constitute a 'Public Health Issue,' NCATS Director Warns

#ERDC2018 – When Treating Rare Disease Patients, Don't Overlook Quality of Life

At Vienna Conference, Gaucher Experts Discuss Treatment of 'Model Disease'

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

Madeline Collin, who has Gaucher disease, with her parents on the day of her graduation from England's University of Bath. (Photo courtesy Madeline Collin)

Rare Disease Day 2018 poster. (Photo courtesy EURORDIS)

A screenshot from the video “In the Land of Rare Disease,” showing a

Rare Case of FAP and Mitochondrial Disease Reported in Study

Dominique Germain, MD, is a Fabry disease expert at the University of Versailles in Paris. (Photo by Larry Luxner)

Madeline Collin, who has Gaucher disease, speaks about the challenges in her life during a March 4 conference in Vienna. (Photo by Larry Luxner)

Paul Melmeyer

Rare Childhood Disorders Pose Significant Impact on Quality of Life of Children and Parents, Study. Rare childhood disorders, including Pompe disease ...

Jill Bonjean of Eurordis at the recent World Orphan Drug Congress USA. (Photos by Larry Luxner)

Max Gomez moderates the event at the New York Genome Center. (Photos by Larry Luxner). Lappalainen noted that rare diseases ...

NORD Fundraiser Sets 7,000-Mile 'Bike, Run or Walk' Goal for 7,000

Human Genome Project Team at NIH Honored for Advancing Research into Genetic Diseases

Diagnostic Delays in Sanfilippo Syndrome Haven't Improved in Nearly 3 Decades in Netherlands,

MDA conference, Pompe Previous: Pompe Disease ...

Yann Le Cam, CEO of Eurordis.

'Rare Impact Awards' Dinner Marks Orphan Drug Act and NORD at 35

FDA Revising 'Draft Guidance' on Developing Treatments for Rare Diseases

Next:Research Points to Protein in Saliva as Possible Biomarker for Sjögren's

midterm panel

Tissue Remodeling, Toxic Molecule Accumulation Responsible for Upper Airway Involvement in MPS, Study Finds

US 'Right to Try' Law Meets with Mix of Praise and Criticism, Including

(Photo by Janine Gelineau/UConn Health Multimedia Services)

Rare Disease Day at NIH, held this year on Feb. 28, exemplifies NCATS' approach to rare diseases research: involving patients as partners throughout the ...

Pin It on Pinterest. Myasthenia Gravis News · Rare Diseases Constitute a 'Public Health Issue,' NCATS Director Warns

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Rare Disease Groups, Patients Differ on 'Right to Try' Bill Before US Congress

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FDA Commissioner Scott Gottlieb

Study Lauds Narrative Group Therapy as Alternative Strategy to Support Carriers of HTT Mutations

MTPA online database

Cushing's Disease Symptoms, Outcomes Different in Older and Younger Patients

Population Bio Links Mitochondrial Disorder to Parkinson's, Keeps Researching Rare Diseases

Ari Zimran, MD

Pilot Newborn Screening Study Planned for Prader-Willi, 3 Other Rare Diseases

More Studies Needed to Characterize Features, Course of MPS III, Review Says

Vienna to Host RARE2019 Meeting on Rare Diseases

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

... Rare Disease Awareness in India. Cinryze Next:Cinryze Prophylaxis Reduces Number, Severity of HAE Attacks in Children, Trial Shows

Avril Daly, CEO of Retina International, is VP of the Eurordis board of directors. (Photos by Larry Luxner)

Richard Pazdur, MD. A number of individuals and organizations will be recognized for their efforts within the rare disease ...

neurodevelopmental disabilities

PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights

... of people with rare diseases. Durhane Wong-Rieger

Dr. Marshall Summar is chairman of the board of directors at NORD. (Photo by Larry Luxner)

Pin It on Pinterest. Huntington's Disease News. Rare Diseases Constitute a 'Public Health Issue,' NCATS Director Warns

Patient in a hospital bed.

Levels of Signaling Molecules in Tears and Saliva Linked to Symptom Severity, Study Says

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

Signals and fragile X syndrome

Resverlogix Apabetalone

#NORDSummit – Angelman Syndrome on Agenda at NORD's Rare Disease Summit

Fabry Disease Unlikely to be Misdiagnosed for RA in Clinical Practice, Study Suggests

Sanfilippo Diagnosis May Lead to Mental Health Issues for Parents of Patients, Study Reports

How to Find a Disease Specialist

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Pin It on Pinterest. Sjogren's Syndrome News · Rare Diseases Constitute a 'Public Health Issue,' NCATS Director Warns

Cope with AS

Those Who Care for Rare Disease Patients Face Burnout, First US Survey Shows

American Kidney Fund Website Now Includes Information on Alport, Other Rare Kidney Diseases

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Bruce Gelb

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Partners Align on Project to Decrease Time to Diagnosis for Rare Diseases

... AIT Therapeutics' New Nitric Oxide Ventilator Safer for Patients and Healthcare Staff, Study Shows ...

... Rain Won't Stop Us from Curing FA! Wishing Away Friedreich's Ataxia; Rare Diseases Constitute a 'Public Health Issue,' NCATS Director Warns

Copies of a report commissioned by NORD on the Orphan Drug Act. (Photo: Larry Luxner)