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Author: Jeannie


NORD and Partners Host FDA, CDC Leaders in Webinar Addressing Rare Disease Community on COVID-19 Vaccines

WASHINGTON, Jan. 26, 2021 /PRNewswire/ — In an effort to provide clear, useful, authoritative information to rare disease patients and caregivers on the COVID-19 vaccines, on January 15 the National Organization for Rare Disorders (NORD®), along...


Metabolism May Play Role in Recurrent Major Depression

Researchers find that metabolites — small molecules produced by cellular activities — appear to predict risk of recurring depression and may be a new diagnostic tool. To read the...


The first patient has been dosed in a Phase I dose escalation study of JOTROL™. 

JUPITER, Fla., Jan. 11, 2021 /PRNewswire/ — Jupiter Orphan Therapeutics, Inc. (“JOT”) a biotechnology company pioneering a novel and disease modifying product named JOTROL™, today announced that the first patient has...


High-Throughput Mitochondria Transfer Device Developed

Researchers from the UCLA Jonsson Comprehensive Cancer Center say have developed a simple, high-throughput method for transferring isolated mitochondria and their associated mitochondrial DNA into mammalian cells. This approach...


NORD Students for Rare Member Provides Her Perspective on “Unlocking the Power of Health Care Data” Summit Panel

Dec. 16, 2020 As a 2020 scholarship recipient, I was able to attend the NORD Rare Diseases and Orphan Products and Breakthrough Summit in October and to hear from inspiring patients,...


Children’s Hospital, Family of Justina Pelletier bring case to court

Justina Pelletier was 14 in 2013 when she landed in a locked psychiatric unit at Boston Children’s Hospital, temporarily a ward of the state. Her parents stood accused of medical child...


Caroline Payne

Though she be but little, she is fierce Caroline Hope Payne was born 8 weeks premature in 2010, weighing only 2 lbs., 4 oz., resulting in a 47 day...


Lillian DeJean

Lillian DeJean was six years old when she was diagnosed with Mitochondrial Disease. Prior to the official diagnosis, however, she had an underlying, overwhelming feeling that something was wrong....


Sheridan Johnston

Sheridan was diagnosed with mitochondrial disease in 2008 after a lifetime of always being “sickly”. Prior to her diagnosis, she was living a relatively normal life (although filled with...


Advocating Responsibly and Communicating Effectively

Mannie Taimuty-Loomis, MA, LBS and Executive Director of the Jonah & The Whale Foundation, Inc. to learn more and discuss: -What is medical child abuse? Munchausen’s by proxy? What...