Rare Diseases
Amicus Therapeutics’ Pompe disease treatment approved for use in UK
August 17, 2023Medical Communications Amicus Therapeutics, MHRA, NICE, Pompe disease, Rare Diseases
Amicus Therapeutics has announced that the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK has granted marketing authorisation …
AstraZeneca’s Alexion partners with Pfizer for rare disease therapies
July 28, 2023Research and Development Alexion, AstraZeneca, Pfizer, Rare Diseases, genomics, rare disease
Alexion, AstraZeneca’s Rare Disease division, has announced that it has entered a definitive purchase and licence agreement with Pfizer. The …
Santhera and Catalyst Pharmaceuticals sign $231m license and collaboration deal
June 20, 2023Medical Communications Catalyst Pharmaceuticals, Rare Diseases, Vamorolone, licence and collaboration, santhera
Swiss specialty pharmaceutical company Santhera and US-based biopharmaceutical company Catalyst Pharmaceuticals have announced that they have signed an exclusive license …
FDA approves ANDA of 20mg generic Nitisinone capsules
June 1, 2023Medical Communications Analog Pharma, Dipharma, FDA, Nitisinone, Rare Diseases
Analog Pharma and Dipharma have announced that the US Food and Drug Administration (FDA) has approved their 20mg nitisinone capsules, …
Boehringer Ingelheim and Ginkgo Bioworks collaborate over $406m undruggable targets deal
May 9, 2023Research and Development Boehringer Ingelheim, Rare Diseases, cell programming, ginkgo bioworks, undruggable targets
German pharmaceutical company Boehringer Ingelheim (BI) and cell programming and biosecurity platform builder Ginkgo Bioworks have announced a partnership to …
Maze Therapeutics and Sanofi sign exclusive worldwide license agreement for Pompe disease treatment
May 3, 2023Sales and Marketing License Agreement, Pompe disease, Rare Diseases, Sanofi, maze therapeutics, treatment
Maze Therapeutics and Sanofi have announced that they have signed an exclusive worldwide license agreement for Maze’s gluogen synthase 1 …
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