RARECast

548 Folgen

1. Addressing the Challenges of Rare Disease Drug Development

   Vom: 7.3.2018
2. Giving Voice to Patients at the FDA

   Vom: 28.2.2018
3. Finding a Rare Friend

   Vom: 21.2.2018
4. Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis

   Vom: 14.2.2018
5. UPenn's Orphan Disease Center Takes a Rare Approach

   Vom: 7.2.2018
6. How Patients Can Accelerate Translational Research in Rare Disease

   Vom: 31.1.2018
7. Notre Dame Center Leverages Commonalities of Rare and Neglected Diseases

   Vom: 24.1.2018
8. GalXC Quest: Dicerna Bets on Its Unqiue Approach to RNAi

   Vom: 17.1.2018
9. Pharnext Takes a Unique Twist on Repurposing Drugs for Rare Diseases

   Vom: 10.1.2018
10. uniQure Seeks to Find Elusive Market Success with Hemophilia B Gene Therapy

    Vom: 3.1.2018
11. Savara Advances Pipeline of Inhaled Therapeutics for Rare Lung Diseases

    Vom: 27.12.2017
12. Why the Microbiome May Provide a Novel Approach to Treating Certain Rare Diseases

    Vom: 20.12.2017
13. FDA Outlines a Collaborative Approach for Pediatric Rare Disease Trials

    Vom: 13.12.2017
14. Why Patient-Centred Outcomes Are Critical to Rare Disease Drug Trials

    Vom: 6.12.2017
15. What the FDA's New Framework Means for Regenerative Therapies

    Vom: 29.11.2017
16. Measuring the Value of Therapies for Ultra-Rare Diseases

    Vom: 22.11.2017
17. Lessons from Pitching Venture Capitalists

    Vom: 15.11.2017
18. Health Canada Scraps Plans for Orphan Drug Framework

    Vom: 8.11.2017
19. New Investment Boosts Rare Disease Drug Accelerator

    Vom: 1.11.2017
20. Understanding Rare Disease Caregiver Needs

    Vom: 25.10.2017