Acute ischemic stroke still represents a public health challenge due to its high incidence and mortality rate. Current treatments may effectively restore the blood flow, but recanalization by opening occluded vessels does not guarantee a favorable prognosis.
Following on from a successful phase IIa proof-of-mechanism study in myasthenia gravis, NMD Pharma A/S has added clinical data on a second neuromuscular disorder, reporting positive phase IIa results for ignaseclant, in both type I and type II Charcot-Marie-Tooth disease.
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.”