Cure Rare Disease has successfully completed a pre-IND meeting with the FDA regarding its investigational gene therapy program for limb-girdle muscular dystrophy type 2I/R9 (LGMDR9).
Heterocyclic compounds acting as potassium channel subfamily T member 1 (KCNT1) and KCNT1 mutant inhibitors have been reported in a Actio Biosciences Inc. patent as potentially useful for the treatment of neurological disorders.
In recognition of the fact that diversity, equity and inclusion (DEI) are necessary prerequisites for precision medicine, the European Academy of Neurology (EAN) announced the launch of a DEI Hub at its 11th Congress, which is being held in Helsinki through June 24. “We know now that when we talk about personalized medicine, we have to understand that talking about stroke, for example, in a woman is different than talking about stroke in a man,” EAN president Elena Moro told the audience at the opening session of the conference.
Actio Biosciences Inc. has synthesized potassium channel subfamily T member 1 (KCNT1) inhibitors reported to be useful for the treatment of Brugada’s syndrome, early infantile epileptic encephalopathy, epilepsy of infancy with migrating focal seizures, epileptic encephalopathy, nocturnal frontal lobe epilepsy, infantile spasm, Lennox-Gastaut syndrome and myocardial infarction, among others.
Novartis AG has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of familial dysautonomia, Huntington’s disease and spinal muscular atrophy.
University of Sydney has synthesized translocator protein (TSPO; PBR) (A147T mutant) ligands reported to be useful for the diagnosis and treatment of neurological disorders.
Biogen Inc. has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of Huntington’s disease and spinal muscular atrophy (SMA).
Alzheimer’s disease (AD) progression involves microglial activation, and restoring or maintaining microglia homeostasis is a therapeutic approach to fight against AD.
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