NEXT: Nicole Boice, Global Genes, Discusses the Future of Rare Disease

November 18, 2019

Nicole Boice, founder of Global Genes, discusses the future of rare disease and the challenges that will need to be addressed in this brief interview. It was filmed during the Global Genes’ 2019 Rare Patient Advocacy Summit in September 2019. It coincided with the release of Global Gene’s report NEXT: Imagining the Future of Rare Disease

Drug Development

FDA Issues Says It Won’t Approve Incyte’s Ruxolitinib Extended Release Tablets to Treat Rare Blood Disorders

Rare Daily Staff The U.S. Food and Drug Administration has issued to Incyte a complete response letter […]

podcast

Addressing the Barriers to Patient Participation in Clinical Trials

A significant obstacle to getting patients to participate in rare disease clinical trials, particularly children, is the […]

Strategy

Government of Canada Improves Access to Affordable and Effective Drugs for Rare Diseases

Rare Daily Staff The Canadian government said it would invest $1.1 billion (CAN$1.5 billion) over three years […]

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Moderna and Generation Bio Enter Strategic Collaboration to Develop Non-Viral Genetic Medicines

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Drug Development

FDA Approves Incyte’s Zynyz for the Treatment of Rare Cancer

Rare Daily Staff The U.S. Food and Drug Administration has approved Incyte’s Zynyz, a humanized monoclonal antibody […]

Drug Development

FDA Grants Orphan Drug and Rare Pediatric Designations for Dyne’s Treatment for Duchenne Muscular Dystrophy

Rare Daily Staff The U.S. Food and Drug Administration has granted orphan drug and rare pediatric disease […]

Finance

Acer Therapeutics Raises $2.7 Million in Registered Direct Offering Priced At-the-Market

Rare Daily Staff Rare disease drug developer Acer Therapeutics said it is raising $2.675 million in a […]

Deals

Quince Gets Unsolicited Acquisition Offer from Echo Lake Capital

Rare Daily Staff Rare disease therapeutics developer Quince Therapeutics confirmed that it received an unsolicited offer to […]

Drug Development

Novartis Reports Long-Term Efficacy of Zolgensma

Rare Daily Staff Novartis presented new data from two-long terms studies of Zolgensma, its one-time gene therapy […]

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NEXT: Nicole Boice, Global Genes, Discusses the Future of Rare Disease

Related

FDA Issues Says It Won’t Approve Incyte’s Ruxolitinib Extended Release Tablets to Treat Rare Blood Disorders

Addressing the Barriers to Patient Participation in Clinical Trials

Government of Canada Improves Access to Affordable and Effective Drugs for Rare Diseases

Moderna and Generation Bio Enter Strategic Collaboration to Develop Non-Viral Genetic Medicines

FDA Approves Incyte’s Zynyz for the Treatment of Rare Cancer

FDA Grants Orphan Drug and Rare Pediatric Designations for Dyne’s Treatment for Duchenne Muscular Dystrophy

Acer Therapeutics Raises $2.7 Million in Registered Direct Offering Priced At-the-Market

Quince Gets Unsolicited Acquisition Offer from Echo Lake Capital

Novartis Reports Long-Term Efficacy of Zolgensma

Stay Connected