A phthalazinone CRBN ligand enables targeted protein degradation to address IMiD side effects and drug resistance in cancer therapy.
A CYP3A inhibitor reduces first-pass metabolism of an OX2R agonist, raising plasma levels and half-life to improve wakefulness treatment.
CDR-optimized humanized anti-NGF antibodies raise affinity and specificity while lowering immunogenicity for safer chronic pain treatment.
An immediate-release stimulant plus extended-release non-dopamine agonist maintains ADHD symptom control while limiting long-term tolerance.
By inhibiting SLC6A19 transport, this case shows a way to control phenylalanine levels in PKU without the limits and adverse events of enzyme therapy.
Combining alpha-7 nicotinic receptor activation with 5-HT3 modulation improves cognitive and negative schizophrenia symptoms while limiting GI side effects.
Combining A19-144 or A2-73 with sub-therapeutic AED doses maintains seizure control while reducing memory loss and AED burden.
Adjusting oral cavity pH to 2-4 enables intraoral ecopipam absorption, improving bioavailability while reducing first-pass metabolites.
IgSF-domain antibodies stabilize TREM2 on the cell surface, prevent ectodomain shedding, and preserve phagocytic and signaling activity.
Small molecules bind SARM1 catalytic residues to reduce NAD+ depletion and help prevent axonal degeneration in neurological disease.
A non-hallucinogenic LSD derivative targets 5-HT2A receptors to reduce alcohol and substance use with fewer side effects.
Intra-cisterna magna AAVhu68 delivery of GLB1 restores CNS β-galactosidase activity and helps reduce seizures and neurocognitive decline.
IgSF-domain antibodies stabilize cell-surface TREM2 and reduce ectodomain shedding, helping preserve phagocytosis and signaling.
Distinct salt and crystalline forms of Compound 1 improve formulation stability and efficacy while preserving T-type calcium channel modulation.
A multi-chain chimeric polypeptide binds TGF-β receptor II to reduce neuroinflammation and help slow disorder progression.
Light-activated bistable opsins targeted to thalamic neurons help suppress seizures with more specific control than drugs or DBS.
A MOG-binding antibody improves brain accumulation after peripheral dosing, addressing blood-brain barrier limits on CNS delivery.
Low-dose IL-2 targets immune dysregulation to prevent ASD during pregnancy and alleviate core symptoms in at-risk children.
A blood and CSF biomarker panel improves Alzheimer's diagnosis, staging, and treatment-response tracking without invasive brain biopsy.
A drug approach using carbasalate calcium targets hydrocephalus while avoiding the infection and complication risks of shunt surgery.
Small-molecule Cdc42 modulators target intersectin signaling to address Alzheimer's pathology, improving cognition and reducing plaques.
Early botulinum neurotoxin dosing can suppress nociceptive and inflammatory agents to reduce acute and chronic TBI symptoms.
Fecal RT-PCR quantifies enterobacteria markers to diagnose mental disease objectively and assess severity without invasive testing.
A Prdx3 peptide composition separates ROS scavenging and mitophagy control to improve mitochondrial quality in disease treatment.
Using lacosamide with oxcarbazepine, this case shows sustained executive dysfunction symptom control with fewer side effects and less tolerance.
Blocking SEMA4D receptor signaling reduces neuroinflammation, promotes myelination, and improves memory, locomotion, and anxiety-related symptoms.
Specific CDR-engineered anti-TrkA antibodies improve TrkA binding and pain relief while avoiding the safety and dose limits of existing inhibitors.
Charged affinity-linked molecules bind abnormal protein aggregates to reduce toxic buildup while aiming to limit side effects in neurodegenerative therapy.
PFA-fixed dendritic cells loaded with hHsp60sp stabilize antigen presentation and restore defective HLA-E-restricted CD8+ Treg function in autoimmune disease.
Transmucosal ALPHA-1062 bypasses first-pass metabolism and GI side effects while enabling preservative-free multi-use TBI delivery.
CDR1 light-chain mutations prevent isomerization, preserving histone 2A/4 binding and NET-inhibiting activity over time.
Combining apilimod with glutamatergic agents helps reduce glutamate excitotoxicity while preserving muscle strength and motor function in ALS.
Six EP4 antagonist polymorphs use controlled crystallization to improve stability, processing, solubility, and bioavailability for drug formulation.
A two-step induction medium turns dental stem cells into dopaminergic neurons in 6 days with high yield and minimal toxicity.
A psychedelic plus 5-HT2A antagonist and optional D-serine can preserve neuroplasticity while limiting hallucinogenic and positive symptoms.
Targeting specific TRPV1 epitopes enables antibodies to block capsaicin-driven pain while minimizing heat-related side effects.
Anti-AXL antibodies bind separate epitopes from Gas6 to inhibit AXL activity and improve anti-tumor effects in cancer models.
STX64 blocks steroid hormone sulfatase to curb protein aggregation with greater specificity and fewer side effects in neurodegenerative disease models.
Imine- and carbamate-based phenethylamine prodrugs improve uptake while reducing nausea, hypertension, and abuse potential.
Peptidomimetic compounds selectively inhibit protein N-terminal methyltransferases to address weak targeted treatment options in cancer.
Targeted amino acid changes in AAV capsids improve CNS payload delivery while helping vectors evade pre-existing antibody neutralization.
Nitrogen-containing heterocyclic compounds target PCSK9 to lower LDL-C orally, avoiding injection-based treatment barriers and cost.
Allogeneic mitochondria delivered after TBI help repair mitochondrial dysfunction, ease post-concussion symptoms, and reduce secondary brain injury.
Monoterpenes such as perillyl alcohol temporarily open the blood-brain barrier to improve CAR-T and drug delivery for CNS tumors.
Targeted capsid mutations improve CNS biodistribution and transduction of dependoparvovirus vectors for payload delivery.
Early IGF-1 and IGFBP-3 dosing stabilizes blood flow in preterm infants, lowering IVH risk and reducing vasopressor use.
Selective GYS1-inhibiting carboxamides lower pathological glycogen stores while limiting broader disruption of glycogen synthesis.
Alpha1-antitrypsin inhibits coronavirus entry and neural inflammation while biomarker screening guides susceptibility and dosing.
Tetrahydroquinoline derivatives inhibit ferroptosis through radical scavenging and iron chelation, offering a new route for ALS treatment.