FDA offers new details on quicker approval pathway for personalized gene therapies
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Japanese pharmaceutical company Shionogi has agreed to acquire Texas-based rare disease biotech IntraBio in a deal valued at $2 billion.
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A $160 million non-profit initiative in Boston aims to revolutionize the development of genetic medicines for rare and ultra-rare diseases.
A woman was hospitalized after experiencing a rare medical condition triggered by extreme happiness following her daughter's wedding.
Vertex Pharma's $10 billion purchase of Crinetics sparks a near‑doubling of its shares and a biotech‑M&A surge
An AI model successfully diagnosed rare diseases in 18 children after doctors were stumped.