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September 15, 2015

TOPIC: Advocacy, Industry

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

Posted at September 9, 2015 04:28 pm by Jennifer Huron

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of rare diseases still without FDA-approved treatments, we have recently released a new resource for drug developers — draft guidance document — designed to help them navigate the difficult and unique challenges of developing and… Read More

December 23, 2014

TOPIC: Featured News, Medical, Patients & Members, Research, Advocacy, Industry, Patient Stories

The Will to Live and the Strength for a Cure

Posted at December 12, 2014 06:15 am by Lisa Sencen

The day Joshua Frase was born, his parents Alison and Paul, were given grave news about their newborn’s life – he most likely would not survive that day. His weakened muscles could not function normally which affected his ability to breathe on his own. Miraculously, Joshua survived, and when he was three-months old, a muscle biopsy revealed he had… Read More

December 23, 2014

TOPIC: Medical, Patients & Members, Advocacy, Industry, Patient Stories

Saving Eliza

Posted at December 12, 2014 06:13 am by admin

I’m honored to be speaking at the NORD Breakthrough Summit on Rare Diseases and Orphan Products in October.   Social media, as I continue to learn more every day, is an always changing, rapidly moving, fickle and funny world.  Trends can get hot and can come and go overnight, and others go on and on (ALS).  Our journey for the… Read More

December 23, 2014

TOPIC: Featured News, Medical, Patients & Members, Industry, Patient Stories

Life is a Special Occasion

Posted at December 12, 2014 06:08 am by Lisa Sencen

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 loves to sing, dance, play guitar and violin, and like most teenagers, enjoys spending time with friends. Alena has a deep-rooted appreciation of life and cherishes every moment –… Read More

December 18, 2014

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

The Will to Live and the Strength for a Cure

Posted at December 12, 2014 05:54 am by admin

The day Joshua Frase was born, his parents Alison and Paul, were given grave news about their newborn’s life – he most likely would not survive that day. His weakened muscles could not function normally which affected his ability to breathe on his own. Miraculously, Joshua survived, and when he was three-months old, a muscle biopsy revealed he had… Read More