RARECast

548 Episodes

1. Addressing the Challenges of Rare Disease Drug Development

   Published: 3/7/2018
2. Giving Voice to Patients at the FDA

   Published: 2/28/2018
3. Finding a Rare Friend

   Published: 2/21/2018
4. Why a Sports Physical May Trigger a Doctor to Think About a Rare Diagnosis

   Published: 2/14/2018
5. UPenn's Orphan Disease Center Takes a Rare Approach

   Published: 2/7/2018
6. How Patients Can Accelerate Translational Research in Rare Disease

   Published: 1/31/2018
7. Notre Dame Center Leverages Commonalities of Rare and Neglected Diseases

   Published: 1/24/2018
8. GalXC Quest: Dicerna Bets on Its Unqiue Approach to RNAi

   Published: 1/17/2018
9. Pharnext Takes a Unique Twist on Repurposing Drugs for Rare Diseases

   Published: 1/10/2018
10. uniQure Seeks to Find Elusive Market Success with Hemophilia B Gene Therapy

    Published: 1/3/2018
11. Savara Advances Pipeline of Inhaled Therapeutics for Rare Lung Diseases

    Published: 12/27/2017
12. Why the Microbiome May Provide a Novel Approach to Treating Certain Rare Diseases

    Published: 12/20/2017
13. FDA Outlines a Collaborative Approach for Pediatric Rare Disease Trials

    Published: 12/13/2017
14. Why Patient-Centred Outcomes Are Critical to Rare Disease Drug Trials

    Published: 12/6/2017
15. What the FDA's New Framework Means for Regenerative Therapies

    Published: 11/29/2017
16. Measuring the Value of Therapies for Ultra-Rare Diseases

    Published: 11/22/2017
17. Lessons from Pitching Venture Capitalists

    Published: 11/15/2017
18. Health Canada Scraps Plans for Orphan Drug Framework

    Published: 11/8/2017
19. New Investment Boosts Rare Disease Drug Accelerator

    Published: 11/1/2017
20. Understanding Rare Disease Caregiver Needs

    Published: 10/25/2017