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Roche to take IONIS-HTTRx pivotal 'quickly' following early efficacy signal in HD

March 5, 2018

Stanley Crooke, chairman and CEO of Ionis Pharmaceuticals Inc., was buoyant on a conference call Friday as the company reported details from the phase I/II study of IONIS-HTTRx (RG-6042) in individuals with Huntington's disease (HD).

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BioWorld MedTech briefs for Jan. 23, 2026.

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Neurotrimin unveiled as marker in intellectual disability

BioWorld Science

Neurotrimin (NTM) is a member of the IgLON family, the disruption of which has been tied to emotional learning deficits and anxiety-like behavior in animal...

DNA mutations or genetic disorder concept art

Biallelic variants in COX18 identified as cause of Charcot-Marie-Tooth disease

BioWorld Science

Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited...

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Cell/gene therapy sector now sustainable; China competition mounting

BioWorld

There was an upbeat message for cell and gene therapy companies in the 2026 industry update presented as the J.P. Morgan Healthcare Conference opened on Monday,...