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Rare Disease

News
Scenesse

Clinuvel gets approval in US for rare skin disorder drug Sce...

News
Sarepta rebounds on data for limb-girdle muscular dystrophy gene therapy

Sarepta rebounds on data for limb-girdle muscular dystrophy ...

News
Young lonely man in a wheelchair on a balcony looking at the nature in the spring

NS Pharma steals a march on Sarepta with DMD drug

News
Zolgensma

Novartis points to Zolgensma long-term benefits amid data sc...

News
Roche preps filings for satralizumab in rare disease NMOSD

Roche preps filings for satralizumab in rare disease NMOSD

Views & Analysis
Who-will-be-the-biggest-pharma-players-in-orphan-drugs

Who will be the biggest pharma players in orphan drugs?

New analysis from GlobalData has identified the predicted top 15 pharma players in 2025 for organ drugs, with Roche coming out in the lead.

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Editor's Picks

  1. Novartis snaps up PI3Kα breast cancer drug in $3bn deal
  2. US court says pharma giants must face False Claims suit
  3. Meningitis outbreak in UK prompts call to widen vaccination
  4. US judge rules Kennedy's vaccine changes 'unlawful'
  5. Generic semaglutide in India, and other weight-loss news
News
Ambrosia's sweet $100m round, and other biofinancings

Ambrosia's sweet $100m round, and other biofinancings

Our latest crop of biofinancings has a nine-figure round for Ambrosia, with Pinnacle Medicines, Immutrin, and Gilgamesh Pharma also raising new funds.

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Sales & Marketing
Using AI-driven synthetic personas to take your insights further
Sponsored

Using AI-driven synthetic personas to take your insights fur...

As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.

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The rights of trademark owners are acknowledged. Website and content copyright © 2009-2026, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – an EVERSANA company
The rights of trademark owners are acknowledged. Website and content copyright © 2009-2026, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – an EVERSANA company