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February 28, 2020

TOPIC: Press Releases, Featured News, Medical, Research

NORD Launches Natural History Study for Undiagnosed Rare Diseases

Posted at February 2, 2020 08:00 am by Valaree DonFrancesco

Washington, DC, February 28, 2020—The National Organization for Rare Disorders (NORD)®, the leading independent nonprofit organization representing over 25 million Americans with rare diseases, today launched the Undiagnosed Rare Disease Registry, a new study to collect de-identified information about hard-to-solve medical cases that will enable researchers to perform analyses at a scale previously not possible.

“With the Undiagnosed Rare Disease… Read More

January 23, 2020

TOPIC: Press Releases, Featured News, Medical, Patients & Members, Research

NORD Joins with Pyruvate Kinase Deficiency Community to Publish Historic “Voice of the Patient” Report

Posted at January 1, 2020 02:29 pm by Laura Mullen

Washington, DC, January 23, 2020–The National Organization for Rare Disorders (NORD)®, the Foundation for Rare Blood Diseases (SZB) and the pyruvate kinase deficiency community together made history on September 20, 2019 with patients and families sharing their experiences directly with the US Food and Drug Administration (FDA) and other key stakeholders, and first-of-its-kind testimony from the heavily impacted Amish… Read More

October 11, 2019

TOPIC: Featured News, Medical

NORD Posts New Addition to Video Library – Pediatric Movement Disorders: Guidance for Parents & Physicians

Posted at October 10, 2019 09:41 am by Laura Mullen

NORD has created a short video to help parents, pediatricians and others recognize early signs of pediatric movement disorders and know what to do if they suspect a child may have one. The video features Mered Parnes, MD, Director of the Pediatric Movement Disorders Clinic at Texas Children’s Hospital in an interview he conducted recently with staff from… Read More

October 10, 2019

TOPIC: Featured News, Medical

Information on current NATPARA® shortage

Posted at October 10, 2019 03:33 pm by Laura Mullen

On September 5, 2019, Takeda issued a recall of NATPARA® (parathyroid hormone), due to rubber particulates originating from the rubber septum of the NATPARA cartridge. As of October 9,  NATPARA® is in shortage.

NORD has reached out to the Food and Drug Administration (FDA) on behalf of patients who are now not able to access the… Read More

September 29, 2019

TOPIC: Featured News, Medical, Events, Patient Stories

Amish Community Testimony

Posted at September 9, 2019 07:33 am by Valaree DonFrancesco

Read at Pyruvate Kinase Deficiency (PKD) Externally-Led Patient Focused Drug Development Meeting (PFDD) Meeting on Friday, September 20.

Written By Debbie Drell, Director of Membership, NORD

According to the NIH, more than 500 affected families have been identified with Pyruvate Kinase Deficiency (PK Deficiency) in the world, but just 140 miles north of where we sit here today, you’ll find a… Read More