Rare diseases

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Eric Luellen and Jonah Comstock interview at AAPS

AI-powered drug discovery is outpacing regulators, with Eric...

During the AAPS National Biotech Conference 2025, pharmaphorum’s Jonah Comstock sat down with Eric Luellen, the founder and CTIO of Turing Biosciences

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Abeona prices rare cell disorder gene therapy at $3.1m

Abeona Therapeutics' Zevaskyn has been approved in the US as a treatment for the rare skin disorder recessive dystrophic epidermolysis bullosa (DEB).

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Belén Garijo, Merck's chair and chief executive

Merck agrees $3.9 billion takeover of SpringWorks

Merck KGaA has reached an agreement with SpringWorks on a $47-per-share takeover deal that values the US biotech at $3.9 billion.

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First drug for rare disease APDS cleared for NHS use

Pharming's Joenja has become the first disease-modifying drug that can be used by the NHS to treat APDS, an ultra-rare immunodeficiency disorder.

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Spruce Bio sows the seeds of its new future

Spruce has bounced back from the failure of its lead drug last year, thanks to a licensing deal with BioMarin for a late-stage Sanfilippo drug.

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Norgine's chief executive Janneke van der Kamp

Norgine on a growth charge again with Theravia takeover deal

Netherlands-based pharma group Norgine has expanded its rare disease portfolio with the acquisition of France's Theravia for an undisclosed sum.

AI-powered drug discovery is outpacing regulators, with Eric...

Abeona prices rare cell disorder gene therapy at $3.1m

Merck agrees $3.9 billion takeover of SpringWorks

First drug for rare disease APDS cleared for NHS use

Spruce Bio sows the seeds of its new future

Norgine on a growth charge again with Theravia takeover deal

Remepy's hybrid Parkinson's therapy poised for phase 3

Discover a new era of healthcare panel engagement: Grounded ...

Rare diseases

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