Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3).
Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with progressive muscle degeneration. There is currently no approved treatment targeting the underlying cause of the disease.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurodegeneration.
Researchers from Massachusetts General Hospital and affiliated organizations have published preclinical data for [18F]CNL-02, a positron emission tomography (PET) radioligand targeting mitochondrial complex I (MC-I) that is being developed for the diagnosis of Alzheimer’s disease and other neurodegenerative disorders.
Researchers from Nanjing Drum Tower Hospital reported findings from their evaluation of the role of NLR family CARD domain containing 5 (NLRC5) in post-stroke neuroinflammation. Immunofluorescence staining of mouse brains revealed that NLRC5 was mainly expressed in microglia.
Navega Therapeutics Inc. has received a $4 million Translational Science grant from the California Institute for Regenerative Medicine (CIRM) to support its work addressing neuropathic pain. The grant will fund the final preclinical development studies of NT-Z001 leading to an IND submission.
Spinal cord injury (SCI) is a condition affecting the central nervous system characterized by both motor and sensory impairment. SCI causes lipid accumulation in the form of lipid droplets, which may contribute to the progression of other diseases.
Researchers from Astrazeneca plc and Karolinska Institutet have described the positron emission tomography (PET) evaluation of [11C]AZD-2423, a chemokine receptor type 2 (CCR2) radioligand in non-human primate (NHP) brain imaging. CCR2 is essential in immune cell migration control. It is mainly expressed in microglia and monocytes and closely linked to the brain’s immune defense.
Researchers from the University College Cork have described the neuroprotective effect of TMP-269, a class-IIa-specific histone deacetylase (HDAC) inhibitor in preclinical models of PD. Pan-inhibitors of HDAC have proven effective in models of Parkinson’s disease (PD) by preventing dopaminergic degeneration.
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
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