November 2024

Huntington Disease Treatment: A Glimpse into the Future

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.
Read More
October 2024

Beyond the Cure: Assessing the Long-Term Gains of Curative Therapies for HIV

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.
Read More
August 2024

Fatigue of Joint, and Muscle Pain: The Struggles of systemic lupus erythematosus (SLE) Patients

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?
Read More
July 2024

Gene Therapy: A Guided Journey for Informed Decision-Making

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.
Read More
February 2024

The ageing challenge for rare disease management

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).
Read More
January 2024

Understanding the Socio-Economic Burden of NASH: A Global Perspective

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.
Read More