The future of cystic fibrosis care: a global perspective

SC Bell, MA Mall, H Gutierrez, M Macek… - The Lancet …, 2020 - thelancet.com
Executive summary The past six decades have seen remarkable improvements in health
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …

Molecular mechanisms of phenotypic variability in monogenic autoinflammatory diseases

I Aksentijevich, O Schnappauf - Nature Reviews Rheumatology, 2021 - nature.com
Monogenic autoinflammatory diseases are a group of rheumatologic disorders caused by
dysregulation in the innate immune system. The molecular mechanisms of these disorders …

Ancient familial Mediterranean fever mutations in human pyrin and resistance to Yersinia pestis

YH Park, EF Remmers, W Lee, AK Ombrello… - Nature …, 2020 - nature.com
Familial Mediterranean fever (FMF) is an autoinflammatory disease caused by homozygous
or compound heterozygous gain-of-function mutations in MEFV, which encodes pyrin, an …

Lack of clear and univocal genotype‐phenotype correlation in familial Mediterranean fever patients: A systematic review

S Gangemi, S Manti, V Procopio, M Casciaro… - Clinical …, 2018 - Wiley Online Library
Familial Mediterranean fever (FMF) is the most common autosomal recessive
autoinflammatory disease. To date, following the isolation of more than 280 MEFV sequence …

Familial Mediterranean fever and COVID-19: friends or foes?

A Stella, M Lamkanfi, P Portincasa - Frontiers in immunology, 2020 - frontiersin.org
Familial Mediterranean Fever (FMF) and COVID-19 show a remarkable overlap of clinical
symptoms and similar laboratory findings. Both are characterized by fever, abdominal/chest …

Autoinflammatory disease with focus on NOD2-associated disease in the era of genomic medicine

Q Yao, E Li, B Shen - Autoimmunity, 2019 - Taylor & Francis
Systemic autoinflammatory diseases (SAIDs) represent a spectrum of genetically
heterogeneous inflammatory disorders. Some SAID-associated genes are located in …

[PDF][PDF] Features predicting colchicine efficacy in treatment of children with undefined systemic autoinflammatory disease: a retrospective cohort study

MC Marques, BH Egeli, H Wobma, C Ribeiro… - Eur J …, 2022 - eurjrheumatol.org
Objective: Patients with undefined systemic autoinflammatory diseases (uSAIDs) are
challenging to manage, as there are no guidelines or recommendations for targeted therapy …

Genetic and clinical features of familial mediterranean fever (FMF) in a homogeneous cohort of patients from South-Eastern Italy

A Di Ciaula, M Iacoviello, L Bonfrate, M Khalil… - European Journal of …, 2023 - Elsevier
Abstract Familial Mediterranean Fever (FMF) is linked with the MEFV gene and is the
commonest among monogenic autoinflammatory diseases, with high prevalence in the …

The lancet respiratory medicine commission on the future of care of cystic fibrosis

SC Bell, MA Mall, H Gutierrez, M Macek… - The Lancet …, 2019 - pmc.ncbi.nlm.nih.gov
The past six decades have seen remarkable improvements in health outcomes for people
with cystic fibrosis (CF), which was once a fatal disease of infants and young children …

From genomes to genomic medicine: enabling personalized and precision medicine in the Middle East

PV Jithesh, V Scaria - Personalized Medicine, 2017 - Taylor & Francis
The MENA region is characterized by a high burden of genetic diseases. A number of
genetic diseases have been characterized for the first time in the region. Reports suggest …