Orphan Designated Drugs

Analyst Comment

ASH 2024 discussions shine spotlight on advances in haemophilia treatments

When a room of physicians at ASH 2024 was asked whether they had prescribed a gene therapy in a commercial context, only a handful said yes.

GlobalData Healthcare

Features

Beyond approval: Patients pursue alternate paths to get rare disease treatments

News

Certa plans pivotal scleroderma trial after Phase II data readout

Data Insights

How orphan designated drugs M&A performed in the pharmaceutical industry in Q3 2024

Data Insights

How orphan designated drugs private equity performed in the pharmaceutical industry in Q3 2024

Data Insights

How did the pharmaceutical industry treat orphan designated drugs in Q2 2024 filings?

News

Mirum expands rare disease portfolio with digestive disorder drugs

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