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Health Canada

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FDA approves Priovant’s Lisraya for dermatomyositis

This is the first oral drug treatment for this rare disease in adults

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Ipsen’s cabozantinib accepted by SMC for adults with advanced neuroendocrine tumours

NETs affect approximately seven per 100,000 people in Scotland

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Ipsen to acquire Memo Therapeutics in deal worth more than €700m

The deal expands the company’s rare disease portfolio

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Ipsen appoints Michelle Werner as Executive Vice President, President of North America

Werner previously held senior roles at Bristol Myers Squibb, AstraZeneca and Novartis

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Lundbeck completes patient randomisation for phase 3 trial for multiple system atrophy treatment

The rare neurodegenerative disorder has no cure and no approved treatments

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Data, Evidence, Education: Tackling the Global Multi‑Trillion‑Dollar Rare Disease Burden

Rare diseases are individually uncommon, yet together they affect an estimated 300 million+ people worldwide and touch the lives of over one billion when families and caregivers are included. For...

Medscape Education

phthalmologist, optometrist checking child, little girl vision looking for problems and caring for eye

Why patient engagement must come first in rare disease trials

Explore why patient engagement is the cornerstone of successful rare disease trials, emphasising the importance of designing studies around the realities of patients' lives to build trust and improve outcomes.

Cuttsy + Cuttsy

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#ShortenTheJourney

For the millions of people living with a rare disease, the path to a diagnosis (if ever received) is often a long and frustrating odyssey. This delay is frequently driven...

Medscape Education

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Ipsen appoints Pierrick Lefranc as Executive Vice President Technical Operations

Lefranc has served at Ipsen since 2013, most recently as Senior Vice President

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Women Leaders Rewriting the Protocols of Rare Disease Care

The true measure of a medical leader is the courage to act when existing systems fail the patient. This is the professional and personal ethos of clinical immunologist Dr. Hilary...

Medscape Education

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Medscape Wins Anthem Award for Rare Disease Education Platform

Platform has reached 2.4 million healthcare professionals worldwide since 2021. Newark, NJ - [November 18, 2025] - Medscape, the leading digital platform for physicians and healthcare professionals (HCPs), has been...

Medscape Education

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Ipsen to acquire ImCheck Therapeutics in a deal worth up to €1bn

The acquisition includes a clinical stage acute myeloid leukaemia treatment currently in phase 1/2 trials

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