RARE Drug Development Symposium: Patient Spotlight & Fireside Chat

December 15, 2020

By hearing Nasha Fitter of FOXG1 Research tell her story, we learn how patients can take ownership of their diagnosis and use natural history data sharing to drive research efforts. David Rich of Ipsen also discusses how patients can help prove their own clinical impact.

Deals

Lisa Kadyk & Aditi Desai from CIRM talk about being at Week in RARE

Lisa Kadyk, PhD, Associate Director of Therapeutics, and Aditi Desai, MPH, CPH, Community Outreach Manager, at CIRM, […]

Diagnosis

September is Newborn Screening Awareness Month

Learn what newborn screenings are, why they are important, how regulations vary by states across the U.S., […]

DEI

Cory Lewis Talks About Advocating For Himself as a Gay Man Living with a Rare Disease

Cory Lewis was diagnosed with Sickle Cell Disease (SCD) when he was six years old. As an […]

Living with a RARE Disease

Effie Parks Applauds Sensory Shopping Hours for Back to School

Effie Parks, Community Engagement Coordinator for Global Genes, RARE mom and host of the Once Upon A […]

Diagnosis

The Not-So-Friendly Skies: Flying When You Have a Rare Disease

Updated to include an article about Beyoncé stepping up to get a fan in a wheelchair to […]

Drug Development

Virtual Partnering Event: IN-PART & Global Genes/RARE-X

During this webinar presented with IN-PART, Karmen Trzupek, Senior Director of Scientific Programs at Global Genes, shared […]

Diagnosis

Why Is Collaboration Important in Rare Therapy Development and Research?

Rare drug development cannot move forward, or move forward quickly without collaboration. The ecosystem works best when […]

Diagnosis

How Can Patient Organizations Be Research Ready?

Panelists from the 2023 RARE Drug Development Symposium discuss how patient advocacy organizations can be research ready, […]

Diagnosis

How Can Patient Advocates Be Involved in Rare Therapy Development?

To answer the question of HOW patient advocates can be involved in rare drug development, we must […]

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RARE Drug Development Symposium: Patient Spotlight & Fireside Chat

Related

Lisa Kadyk & Aditi Desai from CIRM talk about being at Week in RARE

September is Newborn Screening Awareness Month

Cory Lewis Talks About Advocating For Himself as a Gay Man Living with a Rare Disease

Effie Parks Applauds Sensory Shopping Hours for Back to School

The Not-So-Friendly Skies: Flying When You Have a Rare Disease

Virtual Partnering Event: IN-PART & Global Genes/RARE-X

Why Is Collaboration Important in Rare Therapy Development and Research?

How Can Patient Organizations Be Research Ready?

How Can Patient Advocates Be Involved in Rare Therapy Development?

Stay Connected