Introduction Mucopolysaccharidoses (MPS) are a group of rare, inherited lysosomal storage disorders caused by the deficiency of enzymes responsible for degrading glycosaminoglycans (GAGs). These conditions result in progressive accumulation of GAGs in tissues, leading to multi-organ dysfunction. While all MPS types share overlapping clinical manife

Hello, and a warm welcome to MedX, where we bring you the latest news and insights from the world of medicine.  FDA Clears Sepiapterin for Phenylketonuria in All Age Groups The U.S. FDA has approved sepiapterin for the treatment of hyperphenylalaninemia in individuals with phenylketonuria (PKU), including both adults and children aged one month and

Hello, and a warm welcome to MedX, where we bring you the latest news and insights from the world of medicine.  FDA Approves Finerenone for Heart Failure with Preserved or Mildly Reduced Ejection Fraction The US FDA has approved the nonsteroidal mineralocorticoid receptor antagonist finerenone for use in patients with heart failure and mildly reduc

Introduction Depressive disorders are among the leading causes of disability worldwide, with notable variations in prevalence and clinical presentation between sexes. Women are generally at higher risk of depression, but men often present with different symptom patterns. Understanding these sex-based symptom profiles is crucial for developing perso

Introduction: Psychotic symptoms are common in Alzheimer’s disease (AD) and are associated with worsened patient outcomes, including faster cognitive decline and increased institutionalization (1). Treatment remains challenging, as no medications are specifically approved for AD-related psychosis (1). Traditional antipsychotics pose a significant r

Introduction: Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by cognitive decline, memory loss, and impaired daily functioning (1). This debilitating neurodegenerative disorder affects 50 million people worldwide and represents a significant public health challenge, with a growing prevalence due to aging populati

Background Ms. A. R., a 47-year-old teacher, had been living with major depressive disorder (MDD) for over 10 years. Despite trials of multiple antidepressants—including selective serotonin reuptake inhibitors (SSRIs), serotonin and norepinephrine reuptake inhibitors (SNRIs), and augmentation with antipsychotics—she continued to experience persiste

Introduction Narcolepsy Type 1 (NT1) affects both genders equally and significantly impairs daily functioning. Its core features, EDS, cataplexy, sleep paralysis, and hypnagogic hallucinations, result from selective degeneration of orexin-producing hypothalamic neurons. First-line therapies such as modafinil, amphetamines, or sodium oxybate are lim

Background Treatment-resistant depression (TRD) remains a major unmet need, as many patients relapse despite antidepressant therapy. Esketamine, a nasal spray formulation of the NMDA receptor antagonist, offers a new option for long-term maintenance after initial response. This pivotal Phase 3 trial evaluated its efficacy and safety in preventing r