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

In Uncategorized by Caroline HornbyJune 24, 2011Leave a Comment

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, 2011Leave a Comment

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, 2011Leave a Comment

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, 2011Leave a Comment

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, 2011Leave a Comment

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 …

Makena backlash & potential impact on the #RareDisease community

In Uncategorized by Caroline HornbyMay 18, 2011Leave a Comment

Siren Interactive kindly provided this guest blog post from their blog, SirenSong. The publicity surrounding KV Pharmaceutical’s pricing and handling of the drug, Makena, is …

Tourette Syndrome Association and Psyadon Pharmaceuticals to collaborate on #OrphanDrug trial

In Uncategorized by Caroline HornbyMay 16, 2011Leave a Comment

The national Tourette Syndrome Association (TSA) and Psyadon Pharmaceuticals Inc. are pleased to announce their collaboration on a clinical trial to determine the potential efficacy …

FDA grants orphan drug status to Provectus Pharma liver cancer drug

In Uncategorized by Caroline HornbyMay 9, 2011Leave a Comment

The US Food and Drug Administration (FDA) has granted orphan drug status for Provectus Pharmaceuticals‘ cancer drug PV-10 as a treatment for liver cancer. Provectus …

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