Rare Disease News

FDA: Another tool helping developers navigate the difficult road to approval of drugs for rare diseases

Published September 15, 2015 by NORD

Categorized in -

Today, the FDA released a blog post about a new tool for drug developers. The FDA writes, “With the vast majority of rare diseases still without FDA-approved treatments, we have …

Continue reading “FDA: Another tool helping developers navigate the difficult road to approval of drugs for rare diseases”

The Will to Live and the Strength for a Cure

Published December 23, 2014 by NORD

Categorized in -

The day Joshua Frase was born, his parents Alison and Paul, were given grave news about their newborn’s life

Saving Eliza

Published December 23, 2014 by NORD

Categorized in -

I’m honored to be speaking at the NORD Breakthrough Summit on Rare Diseases and Orphan Products in October

Life is a Special Occasion

Published December 23, 2014 by NORD

Categorized in -

Alena Galan is a happy 16-year old girl full of life and wise beyond her years. She can light up a room with her quick-witted charm and energetic personality. She …

Continue reading “Life is a Special Occasion”

The Will to Live and the Strength for a Cure

Published December 18, 2014 by NORD

Categorized in -

The day Joshua Frase was born, his parents Alison and Paul, were given grave news about…

NORD Celebration Highlights the Courage of Children and Adults Living with Rare Diseases

Published December 1, 2014 by NORD

Categorized in -

Join us on May 2015 in Washington DC to celebrate the successes of the rare disease community.

Medical Research Should Be Everyone’s Priority

Published September 25, 2014 by NORD

Categorized in -

It’s truly amazing to hear everyone’s unique personal story on why medical research is important to them. Whether it’s a parent whose son or daughter is living with a rare …

Continue reading “Medical Research Should Be Everyone’s Priority”

Living with Blood Cancer

Published September 16, 2014 by NORD

Categorized in -

Editor’s note: The following blog is being published to highlight Blood Cancer Awareness Month and MPN Awareness Day. I was diagnosed with essential thrombocythemia (ET) at age 16 after …

Continue reading “Living with Blood Cancer”

Children’s Cardiomyopathy Awareness Month Urges Families to #KnowYourHeart this September

Published September 2, 2014 by NORD

Categorized in -

Carmina Taylor of Penllyn, Penn., lost her son, Dorien, to sudden cardiac arrest at age 17

NORD Recommendations for Advancing Drug Discovery, Development, and Delivery

Published June 17, 2014 by NORD

Categorized in -

On behalf of the millions of Americans who have diseases with little or no treatment, the National Organization for Rare Disorders (NORD) has submitted a seven-step plan to the 21st …

Continue reading “NORD Recommendations for Advancing Drug Discovery, Development, and Delivery”