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Important medical research and remarkable case reports worth knowing.

Selected from high-relevance medical literature published in the last 14 days.

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FeaturedClinical ResearchMedical Genetics

Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: results from an open-label extension study.

Ann Am Thorac SocSep 1, 2026Open access

This phase 3 open-label extension study evaluated the long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in 251 adolescents and adults with cystic fibrosis and F508del-gating or…

Why it matters

The findings support the long-term safety and efficacy of ELX/TEZ/IVA in managing cystic fibrosis in adolescents and adults, highlighting its potential to improve lung function and overall health in this population.

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Also worth knowing

Clinical ResearchMedical Genetics

Safety and efficacy of guanabenz in early-childhood onset vanishing white matter: primary analysis of a single-arm, phase 1/2 trial.

This single-arm, phase 1/2 trial evaluated the safety and efficacy of guanabenz in 31 children with early-childhood onset vanishing white matter.

Lancet NeurolSep 1, 2026Read summary
Clinical ResearchNeurology

Prion disease mimicking rapidly progressive Alzheimer disease: case series and systematic review.

Prion disease and Alzheimer disease (AD) are common causes of rapidly progressive dementia (RPD). Although most patients with prion disease are distinguished by MRI and CSF findings, selected cases mimic rapidly…

J NeurolSep 7, 2026Open accessRead summary
Clinical ResearchNeurology

Impact of Baseline Polyneuropathy Severity on Eplontersen Efficacy in the NEURO-TTRansform Clinical Trial.

This post hoc analysis of the NEURO-TTRansform trial evaluated the effect of baseline severity of hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) on the efficacy of eplontersen.

J Peripher Nerv SystSep 1, 2026Open accessRead summary
Clinical ResearchInfectious Diseases

Immunogenicity and safety of prophylactic HPV vaccines in people living with HIV: A systematic review and meta-analysis.

This systematic review and meta-analysis evaluated the immunogenicity and safety of prophylactic HPV vaccines in people living with HIV (PLWH). The analysis included 12 studies with 1,493 participants for HPV16.

Hum Vaccin ImmunotherSep 2, 2026Open accessRead summary
Clinical ResearchMedical Genetics

Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.

This phase 3 randomized controlled trial evaluated the efficacy of DTX401 gene therapy in patients aged 8 years and older with glycogen storage disease type Ia (GSDIa).

J Inherit Metab DisSep 1, 2026Open accessRead summary
Clinical ResearchGastroenterology

The Epidemiology of PRSS1 Hereditary Pancreatitis and Its Clinical Implications: A Systematic Review.

This systematic review examined the epidemiology and clinical implications of PRSS1 hereditary pancreatitis (HP) across 68 studies.

PancreasSep 1, 2026Open accessRead summary
Clinical ResearchNeurology

Treatment of multiple sclerosis with peptide-coupled red blood cells induces antigen-specific T regulatory cells.

This phase Ib clinical trial evaluated the safety and efficacy of peptide-coupled red blood cells (pcRBCs) in patients with multiple sclerosis (MS).

Proc Natl Acad Sci U S AAug 31, 2026Read summary
Clinical ResearchEndocrinology

Growth hormone deficiency: craniofacial characteristics and changes after growth hormone therapy - a systematic review.

This systematic review examined craniofacial characteristics in children and adolescents with non-syndromic growth hormone deficiency (GHD) and the effects of growth hormone therapy (GHT).

EndocrineSep 9, 2026Open accessRead summary
Clinical ResearchMedical Genetics

A systematic review and network meta-analysis of single nucleotide polymorphisms associated with oral submucous fibrosis risk.

This systematic review and network meta-analysis evaluated the association between single nucleotide polymorphisms (SNPs) and the risk of oral submucous fibrosis (OSF) in a population of 2545 cases and 3772 controls.

Medicine (Baltimore)Sep 4, 2026Read summary
Clinical ResearchMedical Genetics

Distribution of CYP2C19 polymorphism and predicted metabolizer phenotypes in the Indian population: A systematic review.

This systematic review and meta-analysis examined the prevalence of CYP2C19 polymorphisms and predicted metabolizer phenotypes in the Indian population.

Indian J PharmacolSep 2, 2026Read summary
Clinical ResearchHematology

Systematic review of machine learning approaches for predicting sickle cell crisis and mortality risk at the climate-health nexus.

This systematic review analyzed 34 studies on machine learning models predicting sickle cell anemia (SCA) crises and mortality, focusing on climate-related factors.

BMC Med Inform Decis MakSep 3, 2026Open accessRead summary

Showing 12 of 17 publications that cleared our clinical screen in the last 14 days.

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