Microglia play a crucial role in interacting with neuronal synapses and modulating synaptic
plasticity. This function is particularly significant during postnatal development, as microglia …

Abstract Arginase 1 Deficiency (ARG1‐D) is a rare urea cycle disorder that results in
persistent hyperargininemia and a distinct, progressive neurologic phenotype involving …

Biocatalysis can be defined as the use of isolated enzymes or enzymes inside cells to
convert substrates into products. The term “biocatalysis” is often used in modern organic …

Clinical trial development in rare diseases poses significant study design and methodology
challenges, such as disease heterogeneity and appropriate patient selection, identification …

Early-onset forms of hereditary spastic paraplegia and inborn errors of metabolism that
present with spastic diplegia are among the most common “mimics” of cerebral palsy. Early …

Background The study determined the safety, pharmacokinetics/pharmacodynamics
(PK/PD), and recommended Phase II dose of BCT-100 for arginine auxotrophic tumours in a …

Macrophages functionally adapt to a diverse set of signals, a process that is critical for their
role in maintaining or restoring tissue homeostasis. This process extends to cancer, where …

Arginine, a conditionally essential amino acid, plays a crucial role in several metabolic and
signalling pathways. Arginine metabolism in the body can be significantly increased under …

Background and aims Arginase 1 deficiency (ARG1-D) is a ultrarare disease with
manifestations that cause mobility and cognitive impairment that progress over time and may …

Summary Background Arginase 1 Deficiency (ARG1-D) is a rare debilitating, progressive,
inherited, metabolic disease characterized by marked increases in plasma arginine (pArg) …