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In Depth 11 Jan 2023

Drug repurposing emerges as viable option for rare disease treatment

Opinion 12 Dec 2022

Artificial intelligence: a new era in rare genetic disease diagnosis

PacBio and Boston Children’s Hospital to study genome to benefit patients

Khondrion’s sonlicromanol produces encouraging results for next stage of study

Gene therapy approval “game changer” in hemophilia B treatment

Khondrion’s mitochondrial diseases treatment producing positive results

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Avanzanite Bioscience looks to commercialize innovative medicines for rare diseases in Europe

A new treatment for tackling epidermolysis bullosa

InfanDx tackles early detection of hypoxic ischemic encephalopathy

A biotech boom manifesting real results in rare pediatric diseases

Centogene launches rare and neurodegenerative disease Biodata Network

In Depth 3 Nov 2022

What’s on the horizon for gene editing-based therapies?

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