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Throughout 2024, Prime HCD has continued to publish research addressing challenges faced globally, across diverse groups, and explored the potential impacts of transformative treatments.

Huntington disease (HD) is a rare, inherited neurodegenerative disorder that progressively affects motor skills, cognition, and behaviour. Currently, there is no cure for HD. Existing treatments can alleviate movement disorders and help manage psychiatric symptoms. However, these treatments do not halt disease progression.

Over the past 30 years, antiretroviral therapy (ART) has transformed HIV into a manageable condition, drastically reducing mortality where access is available. Yet, the disease remains a significant challenge, particularly in low- and middle-income countries. Excitingly, curative therapies (CTx) might be within reach in the next 10-20 years. But addressing challenges related to pricing and financing will be essential for ensuring global access. To address these, we need to understand the economic value of CTx and develop a decision-analytic model to estimate long-term costs, health outcomes, and a value-based price for each country.

SLE can be relentless. An inflammatory autoimmune disease that affects multiple systems within the body. It challenges patients across ethnicities, leaving them grappling with symptoms that disrupt their lives. While SLE predominantly affects black women, it also impacts Asian and Caucasian women. Unfortunately, there’s no magic cure. Instead, treatment focuses on symptom management. But what about the quality of life (QoL) of these patients? How does SLE affect their daily experiences, work, and overall well-being?

Gene therapy (GTx) offers a revolutionary treatment option for patients with haemophilia. It’s a one-time treatment that could potentially provide continuous protection for years. This significant treatment decision warrants a thorough understanding and careful consideration of all relevant information.

Global Prevalence: Sickle Cell Disease (SCD) is a widespread genetic blood order, affecting millions worldwide. The majority of cases are found in sub-Saharan Africa and Southeast Asia.

“A remarkably simple and helpful piece of research.” – Chris Mullin, Chief Economist, UK Department of Health and Social Care

Join us in shedding light on the world of rare diseases, impacting 400 million individuals globally:

In the 2019 paper by Carlo Sabbà et al, rare diseases in the elderly: a new perspective for the specialist in geriatrics, Geriatric Care 2019; volume 5:8769, Carlo Sabbà (and co-authors) highlighted the challenges facing geriatric specialists managing rare diseases (RD).

Non-Alcoholic fatty liver disease (NAFLD) and it progressive form, non-alcoholic steatohepatitis (NASH), represent a growing global health concern. These conditions are charactized by the accumulation of fat in the liver, potentially leading to inflammation, fibrosis, cirrhosis, and even liver failure. Accurate diagnosis and assessment of disease severity are crucial for effective management and resource allocation.