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July 2, 2019

TOPIC: Advocacy, Events, Featured News, Get Involved, Industry, Medical, Patients & Members, Sticky Posts for Homepage

Registration Open for 2019 NORD Rare Summit

Posted at July 7, 2019 09:01 am by Christina Jensen

The time is now

  • To address affordability for patients,
  • To drive progress with a unified voice,
  • To understand the role of advancing technology in rare disease, and
  • To register for the 2019 NORD Rare Summit!

In 2019, we believe The Time is Now to prioritize action in order to develop stronger resources, build better outcomes and find cures for the millions of Americans living with… Read More

May 20, 2019

TOPIC: Advocacy, Featured News

The Honorable Sarah Davis: 2019 Rare Impact Award Honoree

Posted at May 5, 2019 03:28 pm by Christina Jensen

Representative Sarah Davis is a dedicated champion of the rare disease community in her home state of Texas. She is currently serving her fifth term as the State Representative for Texas House District 134, located in Houston, Texas. In Representative Davis’ role as Chair of the Appropriations Subcommittee on Health and Human Services (Article II), she has… Read More

May 20, 2019

TOPIC: Advocacy, Featured News, Get Involved, Patient Stories, Patients & Members

Deborah Skolaski: 2019 Rare Impact Award Honoree

Posted at May 5, 2019 01:31 pm by Christina Jensen

Houston native Deborah Skolaski is the loving grandmother of Cori, a seven year-old living with metachromatic leukodystrophy. MLD is a genetic disorder that affects the nerves, muscles and other organs, as well as behavior. Deborah felt helpless as she observed the struggles that her son, daughter-in-law and Cori were experiencing. However, she was determined to make a… Read More

May 20, 2019

TOPIC: Advocacy, Featured News, Get Involved, Patient Stories, Patients & Members

Madison Shaw: 2019 Rare Impact Award Honoree

Posted at May 5, 2019 01:25 pm by Christina Jensen

Living through a lengthy diagnostic odyssey is unfortunately very common for rare disease patients. Madison “Maddie” Shaw’s journey to diagnosis took place over an intense and frustrating two and a half years when she was 12 and included acute infections, multiple hospitalizations and almost two dozen befuddled doctors. Despite extensive medical attention, it took Maddie’s mom noticing… Read More

May 20, 2019

TOPIC: Advocacy, Featured News, Get Involved

Dr. Debra Regier: 2019 Rare Impact Award Honoree

Posted at May 5, 2019 01:18 pm by Christina Jensen

Debra Regier, M.D., Ph.D. is the Director of Genetic and Genomic Education at Washington, DC’s Children’s National Medical Center, attending physician in genetics and metabolism and Primary Investigator (PI) for the Rare Disease Clinical Research Scholars Program. A graduate of the University of Utah School of Medicine in 2009 as Primary Investigator, Dr. Regier now… Read More