Rare Disease

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.

Digital

To fight rare diseases, win the data battle first

Innovation is the key to evolution. In the pharmaceutical space, brilliant advancements have occurred with drugs with large population pools.

News

Lifeline for Wolman disease kids as NICE backs Alexion drug

Infants with a rare and fatal congenital disorder called Wolman disease in England and Wales will soon have access to a new drug via the NHS that can dramatically extend t

Patients

The rare disease treatment revolution: Pioneering patient-ce...

With over 30 million individuals in the United States alone affected by 7,000 rare diseases,1 it is crucial to provide these patients with timely access to diagnosis and prompt t