MoCD Type A is a rapidly progressive autosomal recessive inborn error of metabolism resulting in toxic sulfite levels causing neurologic sequelae
The hospital has taken a huge step by offering “Titli-palliative care services at home” to provide curative, restorative, and life-prolonging treatment to seriously ill children
Dr. Mona frequently treats children experiencing localized reactions from insect bites and stings.
If approved, Tafinlar + Mekinist may offer a potential new standard-of-care for pediatric patients with this brain cancer
Paxlovid is the first oral therapy specifically designed to combat Covid-19 to be evaluated in a pediatric clinical study
Dr. Peter Houghton will lead the research collaboration for GCCRI and is widely regarded as a leading expert in pediatric cancer research and in the development of novel approaches to treating childhood cancers
Cosentyx is a proven treatment, supported by long-term five-year sustained efficacy and safety data across several inflammatory conditions and with more than 500,000 patients treated worldwide since launch
HYMPAVZI is already approved in the US for patients 12 and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors
A PRV is a tradable voucher granted by the FDA to encourage the development of new treatments for rare pediatric diseases
Venglustat, a glucosylceramide synthase inhibitor (GCSi), works by reducing the abnormal buildup of sugar-and-fat molecules in cells and organs
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