• [[ Whitepaper ]] Connecting With Rare Disease Patient Communities: Patient Advocates

    laureng Whitepapers April 19, 2018
  • [[Whitepaper ]] Disruptive Innovation in Rare Disease Markets

    laureng Whitepapers April 19, 2018
  • Choosing the Right Channel Strategy for Specialty Product Success

    Karina Kusova Clinical Development, Market Access, Orphan Drug Congress, Supply Chain & Manufacturing, Uncategorized, Whitepapers May 3, 2017
  • [Whitepaper] Challenging Conventional Patient Recruitment and Care Strategies for Rare Disease Studies

    Karina Kusova Clinical Development, Orphan Drug Congress, Uncategorized, Whitepapers April 10, 2017
  • [[whitepaper]] The New Reality: Maximizing Value in Rare Disease Launches

    Karina Kusova Clinical Development, Featured on App, Orphan Drug Congress, Uncategorized, Whitepapers April 6, 2017
  • What’s New in Rare Cancer Research? (Spoiler Alert: A Lot!)

    Karina Kusova Advocacy, Clinical Development, Market Access, Orphan Drug Congress, Orphan Drugs Live October 10, 2016

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  • Whitepapers / April 19, 2018

    [[ Whitepaper ]] Connecting With Rare Disease Patient Communities: Patient Advocates

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  • Whitepapers / April 19, 2018

    [[Whitepaper ]] Disruptive Innovation in Rare Disease Markets

    View Post
  • Clinical Development, Orphan Drug Congress, Uncategorized, Whitepapers / April 10, 2017

    [Whitepaper] Challenging Conventional Patient Recruitment and Care Strategies for Rare Disease Studies

    View Post
  • Clinical Development, Featured on App, Orphan Drug Congress, Uncategorized, Whitepapers / April 6, 2017

    [[whitepaper]] The New Reality: Maximizing Value in Rare Disease Launches

    View Post
  • Orphan Drug Congress / November 4, 2016

    Rare Disease: Taking a Different Approach

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#OrphanDrugs and a day of dialogue with NORD, the FDA, and NIH

In Uncategorized by Caroline HornbyJuly 13, 2011

Siren Interactive kindly provided this guest blog post from their blog, SirenSong. It was written by Ciaran Bellwoar, Director of Business Development at Siren Interactive. …

Father, Patient Advocate and Entrepreneur: John Crowley on patient focused clinical trials at the World #OrphanDrug Congress 2011

In Uncategorized by Matt TurnerJuly 13, 2011

John Crowley, CEO, Amicus Therapeutics is set to deliver a unique presentation at this years World #OrphanDrug Congress in Geneva. John’s approach to #clinicaltrialdesign during …

World #OrphanDrug Congress 2011 – Preliminary agenda available

In Uncategorized by Marcia ArdilaJuly 6, 2011

  Following the successful launch of the inaugural event, the World #OrphanDrug Congress 2011 is set to become one of the leading forums for the …

Repligen awarded incentives by US and Europe to develop #OrphanDrug for Spinal Muscular Atrophy

In Uncategorized by Caroline HornbyJune 24, 2011

Repligen announced yesterday that it has been granted Fast Track and orphan drug status by the US FDA and the European Medicines Agency respectively for …

CSL Behring receives EU #OrphanDrug designations

In Uncategorized by Caroline HornbyJune 17, 2011

CSL Behring has been granted Orphan Drug Designations (ODD) by the European Commission. Read the full article from Science 2.0 here. Orphan drug status was …

New discoveries shape future for #OrphanDrugs

In Uncategorized by lauralJune 13, 2011

The subject of orphan diseases is at times a very pessimistic one. These diseases are ones that affect fewer than 200,000 citizens, and although they …

Morphotek cancer drug granted #FDA orphan drug designation

In Uncategorized by Caroline HornbyJune 9, 2011

Morphotek has been granted orphan drug designation by the US Food and Drug Administration (FDA) for their cancer drugs, MORAb-004 (for treatment of sarcoma) and …

Download presentation: The average cost of treatment for the #RareDisease AKU

In Uncategorized by Caroline HornbyMay 20, 2011

The rare disease AKU or Alkaptonuria affects less than one in 250,000 people, but even so care for AKU patients should remain a priority in …

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