Tags » Rare Disease

Guest Blogger: What Have We Achieved So Far In 2014 In Orphan Drugs?

Guest Blogger, Georgia McCollum, is General Manager of Life Sciences at Terrapinn. Here are her thoughts on what has been accomplished for orphan drugs in Europe for 2014. 361 more words

Orphan Drug Development

FDA Breakthrough Therapy Designation #54: Neurocrine Biosciences’ Product For Tardive Dyskinesia

Neurocrine Biosciences, a San Diego pharmaceutical company, announces on October 30th, that the FDA grants the company’s investigational product, NBI-98854, a Breakthrough Therapy Designation (BTD) for the treatment of Tardive Dyskinesia (TD). 156 more words

Rare Diseases

Top 10 Worldwide Selling Orphan Drugs For 2020

EvaluatePharma has published a Report, “Orphan Drug Report 2014”, that evaluates and analyzes global orphan drug trends and developments. The following are several highlights from the Report: 316 more words

Orphan Drug Development

Da série títulos clichês: Quem sou Eu?

Tá bom, vamos lá:

Primeiro que eu não sei se escrevo sobre o que é o blog ou sobre quem sou eu, no entanto, algo já dá para perceber: indecisão faz parte tanto de mim quanto do blog. 338 more words

Doença De Fabry

Fall Updates

No big surprise, but I am constantly struggling to find balance in my life.  As a caregiver who is navigating the rare disease world and ALL that entails, it is hard to find much time for myself (or to cook dinner for that matter). 144 more words


Patient Advocacy: Rare Disease Groups' Use Of Social Media

PatientView, is a UK-based research, publishing, and consultancy group, created out of a belief that “the view of patients should be considered in all important healthcare decisions … formed in response to the emerging powerful new global patient movement.” 205 more words

Rare Diseases

EveryLife Foundation For Rare Diseases: Annual Rare Artist Contest

The EveryLife Foundation for Rare Diseases is “dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.”  The organization works to improve the clinical development process for treatments for rare diseases by collaborating with multiple stakeholders: 260 more words

Rare Diseases