An international, Phase III clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare ...

As the rollout of its recently launched pulmonary arterial hypertension (PAH) drug Winrevair ramps up, Merck is continuing ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

Are you battling a rare disease or think you might be suffering from one? If so, there are resources to get help from ...

Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.

PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...

but the scope of support has expanded as the "Rare Disease Control Act amendment" passed the National Assembly. Ji Young-mi, ...

Katie Coleman, now 33, was diagnosed with a rare type of kidney cancer back in December 2020 after struggling to get doctors ...

For the third quarter of 2024, Ascendis Pharma reported a net loss of €99.2 million, or €1.72 per share (basic and diluted) ...

AstraZeneca’s growth was driven by its portfolio of products in the oncology, biopharmaceuticals and rare disease segments ...

A Brisbane father has died after being diagnosed with an extremely rare and incurable disease that he dubbed the family ...

A European regulatory committee now recommends approval of the Alzheimer’s treatment lecanemab a few months after rejecting ...