Rare Disease

News

At second ask, FDA clears AZ’s Ultomiris for NMOSD

AstraZeneca’s Ultomiris has been approved by the FDA to treat rare autoimmune disease neuromyelitis optica spectrum disorder (NMOSD), six months after an earlier applicati

R&D

The opportunities and challenges facing rare disease therapi...

To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024.

News

Gilead to buy liver disease specialist CymaBay for $4.3bn

Gilead Sciences has beefed up its pipeline with an agreement to buy CymaBay Therapeutics and its seladelpar drug for rare liver disease primary biliary cholangitis (PBC),

Digital

Trust and treatment: GenAI in life sciences in 2024

In 2023, the life sciences industry was rocked by generative artificial intelligence. This year, we'll see some of these ideas become reality.

News

Anavex hit as Rett syndrome drug flunks test

2024 has started on a low for US biotech Anavex Life Sciences, which saw its shares fall sharply this morning after Rett syndrome candidate Anavex 2-73 failed a clinical t

Market Access

NICE and orphan drug recommendations: Should we be more worr...

Leela Barham uses the very latest data – running up to November 2023 – to take stock of NICE’s Single Technology Appraisal (STA) recommendations on orphan drugs versus non-orphans.

At second ask, FDA clears AZ’s Ultomiris for NMOSD

The opportunities and challenges facing rare disease therapi...

Gilead to buy liver disease specialist CymaBay for $4.3bn

Trust and treatment: GenAI in life sciences in 2024

Anavex hit as Rett syndrome drug flunks test

NICE and orphan drug recommendations: Should we be more worr...

RIF job losses are underway at HHS agencies; reports

Health Trends 2025: Reimagining What's Possible

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