Published Date: 05 Nov 2023
Neurofilament light was significantly reduced after receiving vetirisiran and patisiran treatment, suggesting that these drugs may be used as a biomarker of treatment response in patients with hATTR amyloidosis.
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PROPEL will test imeroprubart, an investigational FcRn blocker, against placebo in adults with mild to severe generalized myasthenia gravis.
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New analyses from the D1AMOND clinical program found that 69.8% of participants achieved a clinically meaningful reduction in tic severity within 8 weeks of ecopipam treatment, while an interim 18-month analysis showed sustained improvement without new safety signals.
Full results from the phase 2b LUMA showed that investigational BIIB122/DNL151 did not slow confirmed clinical worsening in early-stage Parkinson's disease compared with placebo.
Early phase 1 findings showed that the PROTAC LRRK2 degrader ARV-102 was associated with improvements in saccadic hypometria and changes in CSF markers of endolysosomal function, neuroinflammation, synaptic integrity, and axonal guidance in participants with Parkinson disease.
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