The month of June is recognized as Scleroderma Awareness Month, a time for spreading awareness about the rare disease scleroderma in the medical community and among the general population. This…
For all the coffee lovers who drink as much coffee as I do each day, this one's for you. Recently, a press release from the Icahn School of Medicine at…
Robbie Elliot’s family, who reside in South Shields, England describes him as a perfect boy with a bubbly personality, always giving, and with a special love of music. According to…
Pfizer’s Regional President for Rare Disease, Reda Guiha, recently offered his opinion to The Parliament magazine about the urgency of what he refers to as the ‘rare disease crisis.’ Pres.…
A recent study investigating treatment for adult acromegaly patients has revealed that switching from injectable somatostatin receptor ligands to oral octreotide leads to an improvement in quality of life and…
Have you ever heard of base editing? This relatively new technology within the field of genome editing offers the potential opportunity to treat a variety of genetic conditions. According to…
ACADIA Pharmaceuticals has released data on NUPLAZID, a selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors. This treatment is meant to treat Parkinson's disease patients, specifically the delusions…
According to a story from bbc.com, five month old Arthur was born with the rare disease spinal muscular atrophy (SMA). He is one of the first people in the UK…
According to a story from MedPage Today, a recent study has determined the familial hypercholesterolemia (FH) can often go undetected on genetic tests that rely on arrays. As an example,…
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