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

In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...

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 ...

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

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

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 ...