NEXT: Luke Rosen, KIF1A.org, Discusses the Future of Rare Disease

November 4, 2019

Luke Rose, founder of KIF1A.org, 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

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NEXT: Luke Rosen, KIF1A.org, Discusses the Future of Rare Disease

Related

Expanding Access to Genome Sequencing in Rural Populations

A Small Molecule Therapy to Regenerate Muscle in People with DMD

Argenx Highlights gMG Drug Data in CIDP

Group Calls for Use of Genome Sequencing as First Line Test for Patients Suspected of Genetic Disease

FDA Grants Fast Track Designation for Lexeo’s Gene Therapy for Friedreich’s Ataxia Cardiomyopathy

Across Healthcare Enters Collaboration with n-Lorem Foundation to Develop Submission Platform for Docs

A 37-Year Path to a Diagnose, but Now a Drug May Be Two Weeks Away

Ultragenyx Reports Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome

Harmony and BioProjet License Potential Treatment for Sleep and Wake Disorders

Stay Connected