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Health Canada

- PMLiVE

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

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

- PMLiVE

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

The rare neurodegenerative disorder has no cure and no approved treatments

- PMLiVE

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

- PMLiVE

#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

- PMLiVE

Ipsen appoints Pierrick Lefranc as Executive Vice President Technical Operations

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

- PMLiVE

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

- PMLiVE

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

- PMLiVE

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

- PMLiVE

Ipsen’s tumour treatment receives MHRA marketing authorisation

This is the first approval in the UK for previously treated advanced neuroendocrine tumours

- PMLiVE

Ipsen receives EC approval for Cabometyx in advanced neuroendocrine tumours

Treatment options following disease progression have previously been limited for many patients

- PMLiVE

Medscape Education Wins SILVER for Rare Disease Campaign

We are thrilled to announce that Medscape Education has been honored with the SILVER Award in the Patient Education Information division category by the 2025 National Health Information Awards! This...

Medscape Education

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