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Infographics by 15 Nov 2021

Gene therapy: Making Rare Disease Therapies Less Rare

Expert Advice 27 May 2020

Why Listening to Patient Organizations is Key in Drug Development

Update: Novartis’ Gene Therapy Zolgensma Gets Nod from EU

Phase III Failure Sinks Drug for Rare Neurological Disorder

Update: FDA Imposes No Penalties for Novartis Data Manipulation Scandal

Ipsen Resumes Phase III Testing for Rare Disease Drug and Abandons Pediatric Trial

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First-in-Class Drug For Inherited Metabolic Diseases Funded with €25M

Interview 5 Feb 2020

Harnessing Genetic Suppression to Treat Rare Disease

More News! 26 Nov 2019

Rare Inflammatory Disease Drug Beats Immunosuppressants in Phase III

More News! 11 Nov 2019

Mereo BioPharma’s Brittle Bone Disease Drug Stumbles in Phase II

€51M Nasdaq IPO Will Fund Centogene’s Rare Disease Diagnostics

UK Firm Raises €51M to Repurpose Drugs for Rare Diseases Using AI

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