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.

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.

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.

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.

Researchers from Alumis Inc. have presented preclinical data for a novel, potential first-in-class tyrosine kinase 2 (TYK2) inhibitor – A-005.

Semaphorin 3A signaling, through the plexin A1/neuropilin 1 (PLXA1/NRP1) receptor complex, is known to disrupt the differentiation and migration of oligodendrocyte precursor cells and mature remyelinating oligodendrocytes. Both semaphorin 3A and plexin A1 are up-regulated in the central nervous system of patients with multiple sclerosis.

Venture capitalists (VCs) on three different panels at Biocom California’s Global Life Science Partnering & Investor Conference gave advice to startups on a range of topics from what they’re looking for before investing to what startups should be doing to find pharma partners.