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In the realm of paediatric haemophilia care, advancements in treatments and the understanding of disease burdens have been a game-changer patients and their families. One noteworthy contributor to this progress is the comprehensive research initiative CHESS PEADS (Cost of Haemophilia in Europe, a Socio-economic Survey). This article explores the significant findings and implications of CHESS PAEDS and discusses the need for further research to shed light on the evolving landscape of paediatric haemophilia care and aid patient access.

Gaucher disease (GD) is a rare lysosomal storage disorder that affects individuals due to a genetic mutation resulting in the dysfunction or absence of the enzyme 'glucocerebrosidase.'

Dementia is an umbrella term covering symptoms affecting memory, movement, and thinking ability. It impacts the way a person speaks, thinks, feels, and behaves. Based on symptoms or causative factors, dementia can be subdivided into several types.

Sickle cell disease (SCD) is a prevalent genetic blood disorder that affects millions of people worldwide. Although it occurs globally, the majority of SCD cases are found in sub-Saharan Africa and Southeast Asia(1). Our article highlights the global burden of SCD, the challenges faced by individuals with the disease, the current treatment landscape, and the importance of raising awareness and improving access to care.

Symptoms include headaches, nausea, vomiting, personality changes, seizures, and cognitive dysfunction. While the exact cause of GBM is not clear, it occurs more frequently in individuals with genetic conditions such as tuberous sclerosis, Turcot syndrome, and neurofibromatosis type I.

Any cancer causes high levels of physical burden and psychological distress to both patients and their caregivers. The bone marrow cancer, Multiple-Myeloma, is particularly burdensome, causing numbness and pain throughout the body.

For several years HCD Economics has keenly watched gene therapy developments in Haemophilia and in 2022 the first two gene therapies, for Haemophilia A & B, were granted marketing authorisation by both the EMA & FDA.

Lysosomal storage diseases (LSDs) are metabolic disorders caused by mutations in genes encoding for the proteins involved in lysosomal homeostasis. Collectively affecting 1:5000 live births and often present as pediatric neurodegenerative diseases.

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive illness of the respiratory system. Tissue in the lungs becomes thick and stiff, forming scars which affects the air sacs, leading to an irreversible decline in lung function. Early symptoms include a gradual onset of shortness of breath, a dry cough and feeling tired. Progression results in numerous associated complications such as pulmonary hypertension, heart failure, pneumonia or pulmonary embolism. 13.5% of IPF patients end up with Lung Cancers.

Informal care is generally thought to mean unpaid care, typically provided to elderly and dependent persons by those they have a social relationship with. Such as a spouse, parent, child or other relative. As well as maybe a neighbour or close friend.