Social cognition (SC) refers to a set of skills necessary for successful social communication and interpersonal relationships. Accumulating evidence points toward a crucial role of the cerebellum in SC. This narrative review summarizes and discusses the social cognitive impairment in cerebellar ataxias (CA), a group of hereditary neurodegenerative diseases of the cerebellum. Substantial impairment in emotion recognition and theory of mind, two essential components of SC, is present in CA. The social cognitive impairment is largely independent of the motor disability, general cognitive deficit, or neuropsychiatric symptoms. Since the impairment reaches comparable levels to autism or schizophrenia, it can substantially impact patients’ quality of life and deserves further attention in future research.
Wednesday, October 4, 2023
Social cognition in degenerative cerebellar ataxias
Simona Karamazovova, Veronika Matuskova, Natalie Svecova, Martin Vyhnalek, Social cognition in degenerative cerebellar ataxias, Current Opinion in Behavioral Sciences, Volume 54, 2023, 101313, doi:10.1016/j.cobeha.2023.101313.
FDA Clears Exploration Trial for Higher Dose of CTI-1601 for Friedreich Ataxia
October 2, 2023. After the company released positive phase 2 data earlier this year, Larimar Therapeutics announced that the FDA has cleared a 4-week, placebo-controlled exploration trial (NCT05579691) assessing its investigational Friedrich ataxia (FA) agent CTI-1601 in doses of 50 mg. The company’s open-label extension (OLE), which will assess 25 mg of CTI-1601 daily, was also cleared for initiation by the FDA.
The OLE, expected to begin in Q1 2024, will include those who completed treatment in the phase 2 dose exploration trial or a prior trial of CTI-1601. In the OLE, investigators will assess safety, tolerability, and pharmacokinetics of the agent, as well as measures of frataxin (FXN) levels and other pharmacodynamic markers. Other objectives include the evaluation of the effects of long-term subcutaneous administration of CTI-1601 on measures of clinical function. Data from the OLE will be compared with a matched set of untreated patients from the Friedreich’s Ataxia Clinical Outcome Measures Study (FACOMS) database.
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