Introduction Chronic migraine is a neurological disorder characterized by headaches on 15 or more days per month, significantly affecting quality of life (1). The calcitonin gene-related peptide (CGRP) pathway is central to migraine pathophysiology, making monoclonal antibodies targeting the CGRP receptor a preventive treatment option (2). Erenumab

Introduction: Acute hepatic porphyria (AHP) comprises four rare inherited disorders caused by mutations in genes encoding enzymes of the heme biosynthesis pathway: acute intermittent porphyria (AIP), hereditary coproporphyria (CPOX), variegate porphyria (PPOX), and δ-aminolevulinic acid dehydratase–deficient porphyria (ALAD) (1). Acute intermittent

Introduction Migraine is a frequent and disabling neurological condition. Preventive therapies are used to reduce the frequency and severity of attacks (1). Currently approved oral preventive medications include divalproex sodium, flunarizine, topiramate, propranolol, and timolol, while injectable options such as CGRP-targeted monoclonal antibodies

Introduction Schizophrenia is a chronic psychiatric disorder marked by hallucinations, delusions, and cognitive deficits, affecting about 1% of the population (1). Traditional antipsychotics mainly target dopamine receptors but often inadequately address negative symptoms and cause significant side effects (2). Xanomeline-trospium chloride, a novel

Introduction Schizophrenia is a chronic psychiatric disorder characterized by positive, negative, and cognitive symptoms, presenting a substantial disease burden and a significant impact on the quality of life and functioning of affected individuals (1). Current antipsychotic therapies predominantly target dopamine D2 receptor antagonism and are as

Introduction: Acute hepatic porphyrias (AHPs) are a group of rare, inherited disorders of heme biosynthesis characterized by potentially life-threatening neurovisceral attacks, chronic symptoms such as fatigue and pain, and long-term complications including renal impairment and hepatocellular carcinoma (1). The disorder arises from partial deficien

Introduction: Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, multisystem disease characterized by neuropathy and cardiomyopathy (1). RNA interference therapy with patisiran, administered at 0.3 mg/kg intravenously every three weeks, has shown durable reductions (~82%) in serum transthyretin (TTR) over 24 months, along with

Introduction Migraine is a severe neurological disorder affecting millions worldwide, often causing intense headache pain with nausea, vomiting, and disability (1). Erenumab is a monoclonal antibody targeting the calcitonin gene-related peptide receptor (CGRP-R), a key pathway in migraine pathophysiology (1). It is administered subcutaneously (SQ)

Hello, and a warm welcome to MedX, where we bring you the latest news and insights from the world of medicine.  FDA Approves Zopapogene Imadenovec for Recurrent Respiratory Papillomatosis The FDA has approved zopapogene imadenovec, a novel adenoviral vector-based immunotherapy, for adult patients with recurrent respiratory papillomatosis. This rare