Express Healthcare4h
Defying the odds: How data and cell & gene therapy are rewriting the rare disease story
Javed Shaikh, Director of RWE & HEOR, Axtria, explains how harnessing next-generation sequencing and AI-powered analytics ...
Gaucher disease: What is it? Doctor explains types, symptoms, treatment
Gaucher disease is a genetic disorder when the body is unable to break down certain fats, leading to fat accumulation in the ...
pharmaphorum7y
NICE recommends Sanofi's Gaucher treatment
England’s NHS will routinely fund Sanofi/Genzyme’s Cerdelga (eliglustat) for the rare condition Gaucher disease after a recommendation from NICE. The cost effectiveness body published final ...
Hindustan Times4d
Gaucher disease: What is it? Doctor explains types, symptoms, treatment
Type 1 (non-neuronopathic): This is the most common form and accounts for approximately 90% of all cases. It primarily affects the spleen, liver, and bone marrow but does not impact the nervous ...