Test could end uncertainty in inherited muscle diseases
A new study suggests long-read 'nanopore' sequencing could transform the diagnosis of inherited diseases and unlock new insights for drug discovery.
AbbVie's Cerevel takeover delivers Parkinson's approval
The first drug from AbbVie's $8.7 billion takeover of Cerevel two years ago, Parkinson's disease therapy Juvmo, has won FDA approval.
MSD, Daiichi pull filing for lung cancer candidate I-DXd
MSD and Daiichi Sankyo's regulatory efforts for their ADCs for cancer have stuttered again, with a withdrawn filing for lung cancer candidate I-DXd.
FDA approves Mirum's oral drug for rare disease FOP
Mirum's new oral therapy for rare bone disease fibrodysplasia ossificans progressiva (FOP) looks set to challenge Regeneron's intravenous Pasatru.
EU clears first drug therapy for rare disease cALD
Neuraxpharm and Minoryx' Nezglyal has become the first approved drug therapy in the EU for rare neurodegenerative disease cALD.
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