The chart below identifies 10 new FDA Products Receiving Orphan Designation for the week of 06/08 – 06/12/15 as of 06/13/15 in ascending “Orphan Drug Designation Date” ...
https://orphandruganaut.wordpress.com/2015/06/13/10-new-fda-orphan-drug-designations-week-of-060815/
The chart below identifies 6 new FDA Products Receiving Orphan Designation for the week of 06/01 – 06/05/15 as of 06/07/15 in ascending “Orphan Drug Designation Date” o...
https://orphandruganaut.wordpress.com/2015/06/07/6-new-fda-orphan-drug-designations-week-of-060115/
This week, GEN (Genetic Engineering & Biotechnology News), publishes an online list of the top 35 Nonprofit Disease Foundations (2015 Edition) that give grants and awards for research. T...
Genzyme, a Sanofi company, announces June 4th that the FDA grants the Breakthrough Therapy Designation (BTD) to its investigational Enzyme Replacement Therapy (ERT), Olipudase Alfa. It is being ...
The CBER BTD #s are current as of 05/31/15 and the CDER BTD #s are current as of 05/29/15. The changes are as follows: • The total # of FDA CDER + CBER BTD Requests received by the FD...
The chart below identifies FDA May 2015 Products Receiving Orphan Designation as of 06/02/15 in ascending “Orphan Drug Designation Date” order. FDA May 2015 Products Rec...
https://orphandruganaut.wordpress.com/2015/06/02/fda-may-2015-products-receiving-orphan-designation/
I – Introduction Friedreich’s Ataxia (FA) is a degenerative neuro-muscular disorder that affects approximately 1/50,000 people in the United States. FA can start anywhere from childhood to a...
https://orphandruganaut.wordpress.com/2015/06/01/friedreichs-ataxia-film-premiere/
The chart below identifies 2 new FDA Products Receiving Orphan Designation for the week of 05/25 – 05/29/15 as of 05/30/15 in ascending “Orphan Drug Designation Date” orde...
https://orphandruganaut.wordpress.com/2015/05/30/2-new-fda-orphan-drug-designations-week-of-052515/
The 1st International Congress on Clinical Genetics and Genetic Counselling in Rare Diseases was held in April 2015 in Seville, Spain. The Orphan Druganaut Blog is honored to have Cristina Payán...
The FDA announces late on May 28th, that Pfizer’s Rapamune (Sirolimus) is approved for the treatment of Lymphangioleiomyomatosis (LAM), a rare and progressive lung disease that affects mostly w...
PTC Therapeutics, a New Jersey biopharmaceutical company, announces this month, that the company is launching a new grant awards program, STRIVE (Strategies to Realize Innovation, Vision, and Emp...
Agilis Biotherapeutics is a biotechnology company focusing on DNA-based therapeutics for rare diseases that affects the Central Nervous System (CNS). The company has formed both academic a...
Rare disease patient families are setting up not-for-profit organizations, leading the way for funding research and accelerating the drug approval process. This Blog Post looks at two r...