I have been living with Pompe disease for almost 34 years. Because this is a genetic disorder that I was born with, I have observed a continuous change throughout my entire life. From a weird-looking ...

Cardiosphere‐derived cell (CDC) infusion was associated with better clinical outcomes at 2 years in patients with single ventricle heart disease. The current study investigates time‐to‐event outcomes ...

Regarding safety, treatment was both safe and well-tolerated by patients with relapsing-remitting MS. A phase 2 trial evaluating Hope Biosciences’ adipose-derived autologous mesenchymal stem cell ...

Some common rare diseases are Haemophilia, Pompe disease, Thalassemia, Sickle-cell Anaemia, and Gaucher’s disease. 2. The Delhi High Court said that the National Rare Diseases Committee (NRDC), which ...

in which the innate immune cells actually contribute to disease pathophysiology. To date, most of our knowledge on trained innate immunity comes from animal models and it is not clear if the human ...

The adaptive immune system develops and learns over time to target specific things that invade the body and cause disease. Sometimes it can mistakenly see the body's own cells as invaders and attack ...

A comprehensive understanding of the long-term behavior and adaptation of these stem cells, particularly in relation to various disease contexts, is vital for optimizing therapeutic outcomes and ...

Living with sickle cell disease is not a proverbial bed of roses; instead, it’s a journey of twists and turns. Yet I’m grateful for the experience. It’s imparted invaluable life lessons that have ...

Microglia, the primary immune cells of the central nervous system ... is a hallmark of neurodegenerative diseases. In Alzheimer's disease, microglia initially clear amyloid-β plaques but ...

This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3 ... in combination with OPFOLDA is indicated for the ...