Tags » Rare Disease

YOUR Vote Matters to the Rare Community!

It’s that time of year again, when the rare disease community looks amongst themselves to identify and nominate agents of change, agents of innovation, and individuals who are working tirelessly on behalf of those affected by one of the 7,000 rare diseases impacting over 350 million people worldwide. 267 more words

Rare Diseases

FDA Breakthrough Therapy Designation Approval Chart (Updated As Of 03/25/15)

This Blog Post updates both the FDA CBER and CDER BTD approval charts as of 03/25/15.

The Total FDA BTD Approval Chart is divided into 2 charts: 413 more words

Orphan Drug Development

FDA Breakthrough Therapy Designation: Statistics Chart Updated (as of 03/20/15)

The CBER BTD #s are current as of 02/28/15 and the CDER BTD #s are current as of 03/20/15.

The changes are as follows:

•   The total # of FDA CDER + CBER BTD Requests received by the FDA increases from 285 to 291… 158 more words

Orphan Drug Development

How Do You Feel?? 

Pain is subjective. No one but you knows how you really feel. When you have RSD, pain is at the core of your treatment and medications. 285 more words


Dear Cowden Syndrome

I would like to share this post with you from my virtual contact: Beating Cowdens.

I couldn’t have said it any better myself!

Rare Disease

Rare Diseases: European Approval Of Stem Cell Therapy

In December 2014, the European Medicines Agency’s (EMA) Committee for Medicinal Products For Human Use (CHMP) adopts a positive opinion recommending a conditional marketing authorization for the medicinal product, Holoclar, a stem cell therapy for a rare disease. 292 more words

Orphan Drug Development