Charcot-Marie-Tooth (CMT) disease is a common neuropathy affecting the peripheral nerves. Compared to Type I, type 2 CMT presents as a slower and more progressive disease tied to axonal dysfunction, accompanied by distal muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.
Phagenesis Ltd. raised $42 million in a series D financing round for its neurostimulation therapy, Phagenyx, which treats swallowing dysfunction. The funds will primarily support commercialization of the device in the U.S. where there is a huge unmet clinical need in dysphagia treatment, Reinhard Krickl, CEO of Phagenesis, told BioWorld.
China State Institute of Pharmaceutical Industry and Jiangsu Nhwa Pharmaceutical Co. Ltd. have jointly described aromatic heterocyclic cyclohexyl aminoalkyl piperidine derivatives acting as serotonin 5-HT1A and/or dopamine D2 and/or D3 receptor agonists reported to be useful for the treatment of Parkinson’s disease.
The long-term use of corticosteroids for treating Duchenne muscular dystrophy (DMD) is tied to several undesired effects, compromising the patient’s quality of life; hence, the use of nonsteroidal drugs is highly desirable in the treatment of DMD. Metriopharm AG is investigating the nonsteroidal drug MP-1032 for DMD and recently presented data from studies in a model of DMD.
Iron-sulfur clusters are co-factors that are involved in several biological processes, such as oxidative phosphorylation, enzymatic reactions, and DNA replication and repair. Scientists from the National Institutes of Health (NIH) have presented a study regarding the clinical manifestations associated with a novel neuromuscular disease gene – CIAO1 – which encodes probable cytosolic iron-sulfur protein assembly protein CIAO1 and is an essential member in the cytoplasmic iron-sulfur assembly machinery.
Pharmaust Ltd.’s monepantel met its primary safety endpoints and showed positive signals of potential efficacy in a phase I trial in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.
On March 4, 2024, several groups of scientists discussed the challenges of investigating the effects of HIV in the central nervous system (CNS) at the oral abstract session on neuropathogenesis of HIV held during the 31st Conference on Retroviruses and Opportunistic Infections (CROI), in Denver. A cure for HIV will require eliminating the virus in all its reservoirs, those tissues where HIV remains latent but retains the capacity for reactivation and replication. However, despite antiretroviral therapy (ART), the virus could continue to replicate continuously at a low level in some reservoirs, including the CNS.
On March 4, 2024, several groups of scientists discussed the challenges of investigating the effects of HIV in the central nervous system (CNS) at the oral abstract session on neuropathogenesis of HIV held during the 31st Conference on Retroviruses and Opportunistic Infections (CROI), in Denver. A cure for HIV will require eliminating the virus in all its reservoirs, those tissues where HIV remains latent but retains the capacity for reactivation and replication. However, despite antiretroviral therapy (ART), the virus could continue to replicate continuously at a low level in some reservoirs, including the CNS.
Suzhou Lexbio Pharm Co. Ltd. has patented macrocyclic triazole derivatives acting as high-affinity nerve growth factor receptor (NTRK1; TRKA) inhibitors reported to be useful for the treatment of cancer, multiple sclerosis, neuropathic pain, Alzheimer’s and Parkinson’s diseases.
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