Jaymin Kang, PharmD

The recommended dose for the treatment of MDD is 42mg once daily with or without food.

Troriluzole is a third generation prodrug of the glutamate modulating agent, riluzole.

Findings showed pegvaliase reduced mean blood Phe levels from baseline by 49.7% compared with 0.3% with diet only at week 72.

Kygevvi contains doxecitine and doxribtimine, both pyrimidine nucleosides.

CTx-1301 is a novel proprietary formulation of dexmethylphenidate, a stimulant that increases norepinephrine and dopamine activity in the brain.

Ulixacaltamide is a highly selective small molecule T-type calcium channel inhibitor designed to block neuronal bursting in the cerebello-thalamo-cortical circuit linked to tremor activity.

Uzedy is a subcutaneous, extended-release formulation of risperidone, an atypical antipsychotic.

After 36 months, patients treated with high dose AMT-130 saw a 75% slowing of disease progression vs the external control.

A phase 1/2 study is currently underway to evaluate SAR446268 in participants aged 10 to 50 years with non-congenital myotonic dystrophy type 1.

Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.

Zilganersen is an antisense oligonucleotide designed to block the production of excess GFAP caused by mutations in the GFAP gene.

Idebenone targets the mitochondrial dysfunction of retinal ganglion cells to prevent vision loss.

Koselugo is an oral, selective MEK inhibitor.

GTx-104 is an novel, intravenous formulation of nimodipine with the potential to lower food effects, drug-drug interactions, dosing errors as well as better manage hypotension.

Evolocumab is a proprotein convertase subtilisin kexin type 9 inhibitor.

The approval for SC injection was based on data from the open-label extension of the phase 3 CLARITY AD trial.

Rilzabrutinib is a small-molecule, reversible, Bruton tyrosine kinase inhibitor.

The extension was needed to review newly submitted long-term clinical data for all patients in the pivotal study following an FDA information request.

The original NDA submission included data from the registration-directed phase 3 MOVE-FA trial as well as 2 long-term studies evaluating vatiquinone in FA.

Approved in 2022, Skysona is indicated to slow the progression of neurologic dysfunction in boys 4 to 17 years of age with early, active cerebral adrenoleukodystrophy.

Sepiapterin is a PAH activator that reduces blood Phe levels by increasing the intracellular concentrations of tetrahydrobiopterin and protecting against PAH enzyme misfolding.

Gefurulimab is a dual-binding nanobody designed to bind to the C5 protein in the terminal complement cascade and prevent the overactivation of the body’s immune system.

FirstLight and RadiantLight evaluated the safety and efficacy of oveporexton for the treatment of narcolepsy with cataplexy in participants aged 16 to 70 years old.

Tividenofusp alfa is a novel central nervous system-penetrant enzyme replacement therapy designed to address the neurological manifestations of MPS II by delivering IDS into the brain and body.

Atogepant is an oral calcitonin gene-related peptide receptor antagonist indicated for the preventive treatment of migraine in adults.

FNP-223 is currently being investigated in a phase 2 trial evaluating the treatment in adults with possible or probable PSP-Richardson syndrome.

The amended titration schedule was based on the randomized, double-blinded TRAILBLAZER-ALZ 6 trial.

Xifyrm alone is not recommended for use when rapid onset of analgesia is required.

TLX101-CDx is an investigational positron emission tomography agent that targets the membrane transport proteins, LAT1 and LAT2.

The Horizon 3.0 TMS Therapy System is a computerized, noninvasive medical device that directs electrical currents at different regions of the cerebral cortex.