In February 2024, when she was five, Liliana was diagnosed with Morquio syndrome — also known as mucopolysaccharidosis type ...

For the first time, Hospital Kuala Lumpur (HKL) is proudly hosting the annual Rare Disease Day 2025 celebration to raise ...

Klinefelter syndrome is a genetic condition that affects only males. Here's what you should know about the causes, symptoms, and treatment options for the condition. Klinefelter syndrome is a genetic ...

But for a small number of children, the infection triggers strange behavioral changes known as PANDAS syndrome. PANDAS stands for pediatric autoimmune neuropsychiatric disorders associated with ...

Cushing syndrome occurs due to abnormally high levels of cortisol, which is commonly related to steroid use. Symptoms include weight gain, thinning skin, fluid retention, and stretch marks ...

Some forms, like MPS I (Hurler syndrome) and MPS II, also involve severe neurological decline, while others, such as MPS IV (Morquio syndrome), primarily affect the skeletal system without ...

Imposter syndrome is when you doubt your own skills and successes. You feel you're not as talented or worthy as others believe, and you're scared that one day, people will realize that.

Vimizim in Morquio A syndrome and Brineura in CLN2 disease should also drive growth. BioMarin's highest-potential drug, Voxzogo, launched in late 2021. The drug has shown the ability to restore ...