Tags » Rare Disease

FDA January 2015 Products Receiving Orphan Designation

The chart below identifies FDA January 2015 Products Receiving Orphan Designation as of 01/30/15 in ascending “Orphan Drug Designation Date” order.

FDA January 2015 Products Receiving Orphan Designation… 447 more words

Orphan Drug Development

Orphan Drugs And Rare Diseases: 22% of FDA CDER 2014 Approvals Are Personalized Medicines

The Personalized Medicine Coalition’s (PMC) mission is:

“ … to educate federal and state policymakers and private sector health care leaders about personalized medicine, helping them understand the science, the issues and what is needed for the positive evolution of personalized medicine – as critically important. 354 more words

Orphan Drug Development

MPS: In The News

This Blog Post will review recent developments for the rare disease Mucopolysaccharidosis (MPS).

I – Shire’s FDA Fast Track Designation For MPS II (Hunter Syndrome) 659 more words

Orphan Drug Development

FDA Breakthrough Therapy Designation: Statistics Chart Updated (as of 01/23/15)

The CBER BTD #s are current as of 12/31/14 and the CDER BTD #s are current as of 01/23/15.

The changes are as follows:

•    The total # of FDA CDER + CBER BTD Requests received by the FDA increases from 269 to 273… 158 more words

Orphan Drug Development


I like a challenge, especially when the challenge is for a good cause.

What is #Hope4KB?

  1. #Hope4KB is a T-shirt campaign designed to raise awareness for rare diseases around the world through social media (KB is our daughter, Katherine Belle, a three-year-old from Lexington, KY, who is battling an unknown, progressive rare disease);
  2. 191 more words
Rare Disease

27/365 - Last day off

Tomorrow I begin my new career – the delay is over and I can’t wait to get started. No more daytime exploration of places like Somerset House (pictured above). 388 more words

Myasthenia Gravis