Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial pneumonia with poor prognosis and limited therapeutic options. Despite the introduction of pirfenidone and nintedanib, treatment discontinuation due to adverse effects remains common, highlighting the need for better-tolerated, mechanistically distinct agents. Jascayd (nerandomilast), a first-in-class selective phosphodiesterase-4B inhibitor developed by Boehringer Ingelheim, was approved by the FDA in October 2025 for adults with IPF. By elevating intracellular cyclic adenosine monophosphate (cAMP) in inflammatory and structural lung cells, nerandomilast suppresses pro-inflammatory cytokines and fibroblast activation, producing anti-inflammatory and antifibrotic effects with improved tolerability over nonselective PDE4 inhibitors. In phase 3 FIBRONEER-IPF and FIBRONEER-ILD trials, nerandomilast significantly reduced the annual rate of forced vital capacity decline versus placebo, regardless of concomitant antifibrotic use. Common adverse effects included mild diarrhea, nausea, and fatigue, with low discontinuation rates. Long-term extension data demonstrate sustained safety and lung function preservation. Offering both monotherapy and combination potential, Jascayd establishes a new therapeutic class that targets inflammation and fibrosis concurrently, representing a pivotal advancement toward personalized, multi-pathway treatment in IPF.

Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by immune-mediated platelet destruction and impaired platelet production, leading to bleeding risk, morbidity, and reduced quality of life. Incomplete responses, adverse effects, and relapses are the limitations of current treatments, which include corticosteroids, intravenous immunoglobulin, rituximab, and thrombopoietin receptor agonists. On 29 August 2025, the U.S. Food and Drug Administration (FDA) approved Wayrilz (rilzabrutinib), a first-in-class oral Bruton’s tyrosine kinase inhibitor, for the treatment of adult patients with chronic or persistent ITP who have had an insufficient response to previous treatments. Through modulation of both Fc receptor and B-cell receptor signaling, the agent aims to address disease pathophysiology as a treatment that is disease-modifying in nature rather than simplistically dealing with symptoms. Clinical trials, including the key LUNA 3 trial, have shown good efficacy and safety, with durable platelet responses and minimal severe adverse events. This editorial examines rilzabrutinib’s scientific justification, clinical data, comparative analysis, side effects, financial ramifications, and potential applications in the treatment of ITP.

Hypertension is a global health issue, particularly in low- and middle-income countries. Effective management requires medication adherence, which is often compromised in conflict zones such as Sudan. This study examines the relationship between patients’ knowledge of hypertension and their adherence to antihypertensive therapy in Sudan amidst ongoing conflict. A cross-sectional study conducted across six primary healthcare centers in Al-Dammer, Sudan, included 389 hypertensive patients selected through convenience sampling. Data were collected using face-to-face questionnaire that assessed sociodemographics, medication adherence (GMAS), and hypertension knowledge (HKT). Analyses were conducted in SPSS, with percentages for categorical data, means ± SD for continuous data, and nonparametric tests (Shapiro-Wilk, Kruskal-Wallis) for non-normal distributions. Most participants were female (63%), and 43.7% were over 60 years old. Knowledge levels were categorized as average (57.8%), high (25.2%), and low (17%). Adherence rates were as follows: 1% poor, 2.8% low, 22.6% partial, 26.5% good, and 47% high. A positive correlation was found between knowledge and adherence (r = 0.47, p < 0.001). Major barriers to adherence included affordability (43.4%) and unavailability of medications (36.2%). While 82.3% of participants had a history of controlled hypertension, only 58.4% remained controlled at their most recent measurement, with 41.6% presenting uncontrolled blood pressure, highlighting challenges in sustained hypertension management. Improved adherence was associated with higher income, better education, and controlled blood pressure. Higher hypertension knowledge was significantly associated with better adherence and improved blood pressure control. However, sustained control remains a challenge, as 41.6% of participants had uncontrolled hypertension at their last measurement. Addressing economic barriers and medication shortages through targeted interventions is essential for improving long-term hypertension management in conflict settings.

Phenylketonuria (PKU) is a rare metabolic disorder caused by deficient phenylalanine hydroxylase, leading to toxic phenylalanine buildup and severe neurodevelopmental consequences if untreated. Despite advances in newborn screening and dietary management, treatment options remain limited and burdensome. The recent FDA approval of Sephience (sepiapterin) marks a major advancement in PKU care. Sepiapterin, a precursor of tetrahydrobiopterin (BH₄), enhances enzyme activity and stability, offering therapeutic benefit even in patients unresponsive to BH₄ alone. Results from the pivotal APHENITY Phase III trial demonstrated a substantial 63% mean reduction in blood phenylalanine and significant dietary liberalization, with 97% of participants able to increase natural protein intake safely. Importantly, 43% of prior non-responders to sapropterin showed clinical improvement, highlighting its potential to address key gaps in PKU management. With broad approval for children and adults, Sephience provides a disease-targeted therapy that extends beyond dietary restriction. While careful monitoring for adverse effects such as gastrointestinal symptoms and hypophenylalaninemia is required, this approval represents a transformative step in precision medicine. Sephience has the potential to redefine the standard of care and improve long-term quality of life for individuals living with PKU.

Background and Aims: Non‐communicable diseases (NCDs) cause about 70% of global deaths, and early detection through routine medical checkups (RMCs) is a key preventive measure. Despite their importance, RMC uptake remains low, particularly among youth. This study assessed the knowledge, practices, and perceptions of RMCs among medical students in Karachi, Pakistan, and identified barriers to their participation. Methods: A descriptive cross‐sectional study was conducted from May to July 2025 across four medical institutions in Karachi, including JSMU, DUHS, KMDC, and SMBBMC. A total of 246 MBBS students from third to final year were selected via non‐probability convenience sampling. Ethical approval was obtained from JSMU′s Institutional Review Board (IRB No. JSMU/IRB/2025/1025). Data were collected through a structured, pilot‐tested electronic questionnaire assessing independent variables (age, gender, year of study, knowledge, attitudes, and perceived barriers) and outcome variables (practice of RMCs). Cronbach′s alpha for knowledge and practice domains was 0.76 and 0.72, respectively. Data were analyzed using SPSS v26, employing descriptive statistics, independent t‐tests, and ANOVA at a significance level of p < 0.05. Results: Among 246 respondents, 83% were female and the majority (77.2%) were aged 21–23 years. While 90.2% correctly identified the preventive purpose of RMCs, only 21.4% personally scheduled checkups when asymptomatic. Most students (89.9%) supported including mental health screenings in RMCs. Female gender, age group, and year of study were not significantly associated with knowledge scores ( p ≥ 0.05). Major barriers included lack of awareness (27.2%), financial constraints (25.8%), and fear of discovering illness (16%). Although 80.4% had recommended RMCs to others, only 26.8% had undergone a checkup within the past year. Conclusion: Despite good theoretical knowledge and positive attitudes toward RMCs, actual practice among medical students remains poor. Financial barriers, perceived invulnerability, and time constraints hinder participation. Integrating preventive health practices into the curriculum, along with awareness campaigns and affordable services, may bridge the knowledge–practice gap.

Human immunodeficiency virus type 1 (HIV-1) remains a global health challenge, affecting over 38 million people as of 2024. The recent FDA approval of lenacapavir (Yeztugo®) on 18 June 2025 marks a significant advance in prevention and treatment. Lenacapavir is the first-in-class, long-acting HIV-1 capsid inhibitor, administered twice yearly via subcutaneous injection for pre-exposure prophylaxis in adults and adolescents ≥35 kg. It targets the capsid protein, disrupting multiple stages of the viral lifecycle without cross-resistance to existing antiretrovirals. Clinical trials (CAPELLA, CALIBRATE) demonstrated robust efficacy, with up to 94% viral suppression when combined with active agents and sustained CD4+ gains. Its prolonged half-life, minimal drug–drug interactions, and convenient dosing improve adherence, privacy, and healthcare access – critical in high-incidence, resource-limited settings. Ongoing PURPOSE trials are evaluating its role in high-risk populations. Lenacapavir’s favorable safety profile and public health potential support its integration into evolving HIV management and prevention strategies, potentially accelerating progress toward epidemic control.