Shenzhen Hanlin Biomedical Technology Co. Ltd. has identified new urolithin A derivatives. They are reported to be useful for the treatment of neurological disorder, aging, muscle disorder and metabolic diseases.
Work at Alicorn Pharmaceutical Co. Ltd. has led to the synthesis of new sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers. They are reported to be useful for the treatment of pain, respiratory and neurological disorders.
Connecta Therapeutics SL has discovered 3-phenoxy-3-phenylpropanamine derivatives described as potentially useful for the treatment of fragile X and Rett syndrome.
Researchers from the University of São Paulo (Brazil) first proposed using eccentric training as a promising intervention to address musculoskeletal impairments associated with Marfan syndrome. Eccentric training is a form of resistance exercise that focuses on muscle lengthening under load and can induce robust skeletal muscle adaptations, including the attenuation of muscle wasting, promotion of myofiber hypertrophy and stimulation of satellite cell activation and proliferation, as previously demonstrated.
Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disorder with unknown etiology, leading to loss of upper and lower motor neurons, muscle paralysis, and, eventually, death. In the majority of cases (about 90%), the disease is sporadic, while the rest have a genetic component.
Trace Biosciences Inc. has obtained IND approval from the FDA for LGW16-03 (Nervetrace Dx), the company’s first nerve-specific fluorescent imaging agent. The IND clearance enables Trace to initiate a first-in-human study evaluating the safety and intraoperative performance of LGW16-03 in surgical settings.
Quince Therapeutics Inc. is scrapping work with dexamethasone sodium phosphate encapsulated in autologous erythrocytes (eDSP) for patients with ataxia-telangiectasia (A-T), and Boral analyst Jason Kolbert said “the core value driver has been effectively removed, and the equity appears fully impaired at this stage.”
Humanwell Pharmaceutical US Inc. has disclosed prodrugs of somatostatin SST4 receptor agonists described as potentially useful for the treatment of visceral and neuropathic pain.
DFNB16, a recessive form of mild-to-moderate human deafness, is caused by mutations in the STRC gene, which encodes stereocilin, a protein essential for the effective function of outer hair cells as cochlear amplifiers. Researchers from the Institut Pasteur and Sorbonne Université in Paris, France, have developed and evaluated the efficacy of an Strc gene therapy in a mouse model of DFNB16.
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