Tags » Rare Disease

6 New FDA Orphan Drug Designations: Week of 05/18/15

The chart below identifies 6 new FDA Products Receiving Orphan Designation for the week of 05/18 – 05/22/15 as of 05/22/15 in ascending “Orphan Drug Designation Date” order. 109 more words

Orphan Drug Development

Sanfilippo Syndrome Gene Therapy: PlasmaTech Biopharmaceuticals’ Rare Pediatric Disease Vouchers

PlasmaTech Biopharmaceuticals, a Dallas-based biopharmaceutical company focusing on protein biologic therapies and oncology supportive care products, announces on May 20th, that the FDA has granted Rare Pediatric Disease Designations to both of their investigational Gene Therapy products for Sanfilippo Syndrome (SF), MPS IIIB and MPS IIIA: 221 more words

Orphan Drug Development

On my way

  • I’m on my way to the next P.T. appointment this morning.  Little bit of apprehension.
  • Went to another FORCE meeting this weekend.  Sigh.  I can’t believe I’m…
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Rare Disease

Europe’s May 2015 Products Recommended For Orphan Drug Designation

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting May 12 – 13, 2015.

At this meeting, there are 11 positive opinions recommending the following medicines for designation as orphan medicinal products. 248 more words

Orphan Drug Development

Visible Differences and the #ToyLikeMe Movement

A few years ago, I was part of a group in charge of choosing teddy bears to sell at the Moebius Syndrome Conferences. We found that it was actually really, really difficult to find a non-smiling teddy bear! 193 more words

Important Announcement: New Leadership

Hello everyone!

As of today, May 19th, 2015, Cynthia Prentice will now be taking over Dercum’s Disease Resources!  Many of you are already familiar with Cynthia from her avid involvement with Dercum’s Disease awareness, through Facebook, or from the early days of communicating about DD via “forums” on the internet.   149 more words

Adiposis Dolorosa

FDA Breakthrough Therapy Designation: Statistics Chart Updated (as of 05/15/15)

The CBER BTD #s are current as of 04/30/15 and the CDER BTD #s are current as of 05/15/15.

The changes are as follows:

•   The total # of FDA CDER + CBER BTD Requests received by the FDA increases from 300 to 304… 158 more words

Rare Diseases