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In Depth 22 Jul 2022

The coming of age story of RNAi technology

Almirall launches new call to find innovative therapies for skin diseases

Biotechs Carve out Niches in Cystic Fibrosis Treatment Space

Emerging Startups Take Aim at Lysosomal Storage Disorders

Targeted Drugs for Rare Autoimmune Disease Hit Regulatory Milestones

Infographics by 15 Nov 2021

Gene therapy: Making Rare Disease Therapies Less Rare

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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

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

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