• [[ 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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Understanding the Unique Challenges and Opportunities in Rare Disease Drug Development

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaFebruary 13, 2017

Nicole Sweeny, Shire joined us at the World Orphan Drug Congress USA 2016 to deliver her presentation on “Understanding the Unique Challenges and Opportunities in Rare …

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Tackling the Rare Diseases therapy deficit –the nonsense mutation read-through agent

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaFebruary 6, 2017

Mark Rothera, PTC Therapeutics, long time friend of the World Orphan Drug Congress USA, joined us once again in 2016. His presentation “Tackling the Rare …

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Adaptive Biomedical Innovation: Innovating How We Innovate

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaJanuary 30, 2017

Rare Disease research is innovative. The Orphan Drug industry has always embraced the NEW. This is why at the World Orphan Drug Congress USA, we …

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Maintaining a relationship based on trust: finding common goals with a KOL and its institution

In Orphan Drug Congress, Orphan Drugs Live, Partnering & Investment by Karina KusovaJanuary 23, 2017

In a multi-stakeholder industry such as that of Orphan Drugs, communication is vital to ensuring strong, effective working relationships. Charles Stark from Emmaus Lifesciences joined …

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Delivering Hope – A First-In-Class Orphan Drug Comes to Market

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaJanuary 19, 2017

Blogged on behalf of ClinicalMind >> Hope is perhaps the one thing that 30 million Americans living with a rare disease have to hold on …

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‘Significant Benefit’ in the European Regulatory Framework for Orphan Medicinal Products

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaJanuary 16, 2017

Orphan Drug regulation has changed over the past 30 years. New legislation like Prescription Drug User Fee Act (PDUFA VI) and 21ST Century Cures are …

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Creating Medicines with Real Value for Orphan Diseases

In Advocacy, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaDecember 19, 2016

We were thrilled to attend the RareVoice Awards in November 2016, where we met up with our Everylife Foundation colleagues to celebrate the amazing work …

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Re-engineering the process rather than new legislations: Europe’s approach to get more, better, faster, cheaper orphan drugs

In Market Access, Orphan Drug Congress, Orphan Drugs Live by Karina KusovaDecember 12, 2016

Despite great progress, scientific advances are not translated into approved therapies fast enough to cater to the patietns in need. Today’s regulatory system, either for MA …

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