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May 20, 2019

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

Donna Appell, R.N.: 2019 Rare Impact Award Honoree

Posted at May 5, 2019 12:27 pm by Christina Jensen

When Donna Appell’s one-year-old daughter Ashley exhibited frequent and unexplainable bruising, Donna turned to her toddler’s pediatrician for answers. She was told that the bruising was “easier to see” because of Ashley’s albinism. After leaving the pediatrician feeling patronized, Donna sought answers herself, learned about a rare bleeding disorder associated with albinism and soon met with a… Read More

May 7, 2019

TOPIC: Advocacy, Featured News, Industry, Patients & Members, Press Releases, Sticky Posts for Homepage

NORD Releases Principles for Assessing Proposals Designed to Lower the Cost of Prescription Drugs in the United States

Posted at May 5, 2019 09:42 am by Christina Jensen

Washington, DC, May 7, 2019 – The National Organization for Rare Disorders (NORD)Ⓡ, the leading independent nonprofit organization representing the 25-30 million Americans living with rare diseases, has announced its development of key drug pricing principles, created with the needs of the rare disease community in mind.

People living with rare diseases must be able to afford the therapies that… Read More

March 18, 2019

TOPIC: Advocacy, Featured News, Get Involved

Orphan Drug Act Resolution Introduced in the House of Representatives

Posted at March 3, 2019 04:05 pm by Christina Jensen

Today, Representatives GK Butterfield and Gus Bilirakis introduced a resolution that celebrates the success of the Orphan Drug Act (ODA) and calls for continued support of the legislation.

Now in its 36th year, the ODA has successfully encouraged the pharmaceutical industry to develop therapies for those with rare diseases. Prior to the ODA’s enactment in 1983, only 34 therapies for… Read More

February 26, 2019

TOPIC: Advocacy, Get Involved, Patient Stories, Rare Disease Day

Ben’s Story in Honor of Rare Disease Day

Posted at February 2, 2019 09:45 am by Christina Jensen

The following story was submitted by Leah Schust in honor of Rare Disease Day. In this story, Leah shares her family’s journey of searching for and receiving a diagnosis for their son, Ben, of  SCN2A Disorder.

Share your story for Rare Disease Day here.


Benjamin, known to all who love him as Ben, is an adorable… Read More

February 25, 2019

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

Connor’s Story in Honor of Rare Disease Day

Posted at February 2, 2019 01:06 pm by Christina Jensen

The following story was submitted by Katia Luedtke in honor of Rare Disease Day. In this story, Katia shares her family’s journey of searching for and receiving a diagnosis for their son, Connor, of Snyder-Robinson Syndrome (SRS). 

Share your story for Rare Disease Day here.


Our son Connor was diagnosed with Snyder-Robinson Syndrome (SRS) in… Read More