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Sanofi’s first treatment in the U.S. approved for adults and children with ASMD

Emactuzumab designated as an orphan medicinal product for TGCT in Europe

Amgen announces top-line results from biosimilar phase 3 study

Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome

Vaderis emerges from stealth to start HTT trial

Emtora to use $16.9M grant to develop treatment for rare genetic disease

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Aeglea BioTherapeutics’ arginine 1 deficiency treatment gets EMA ok

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

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