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.
A male patient who harbored a deletion in the intron 7-exon 8 region of the dystrophin gene, DMD, was showing symptoms that matched with Becker muscular dystrophy.
Vivoryon Therapeutics NV said it will conduct an in-depth analysis after reporting top-line data showing the phase IIb European Viviad study testing its oral small molecule, varoglutamstat, in patients with early Alzheimer’s disease missed statistical significance on the primary endpoint measuring changes in cognition over time. Investors, however, opted for a more immediate reaction, sending company shares (Euronext Amsterdam:VVY) plunging 90% March 4, to close at €0.80 (US87 cents)