COVID-19 has caused long-term, ongoing symptoms for some patients. Up until recently, these individuals had been called "covid long haulers." Anthony Fauci, during a White House briefing, has recently changed…
An estimated 13-20 out of every 100,000 people around the globe have idiopathic pulmonary fibrosis (IPF). The condition often has a poor prognosis (3-5 years following diagnosis) and there are…
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Rare diseases are much more common than people think. The word "rare" is off-putting. But the word only pertains to each individual rare condition. Collectively, rare diseases are quite common.…
First of all, a little about me. I’m an Australian, living in the Netherlands with my German husband and our 6-year-old, identical twin daughters. We’re a pretty average family, living…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
As the pandemic shifted cocktail hour onto zoom and professors posted their full course material online, many rare patients reveled. Not because we weren’t suffering through the pandemic too, but…
Emflaza (deflazacort), a corticosteroid, was originally approved by the FDA in 2017 to treat Duchenne muscular dystrophy (DMD) in patients five years or older. This approval was expanded in 2019…
In a recent press release, biopharmaceutical company Aeterna Zentaris Inc. shared that the company would be developing a new therapy for patients with hypoparathyroidism. The preclinical development program will focus…
Genomic testing is a great way to learn not just about your genes, but how your genes impact your health. In support of the Phase 2 CRESTONE clinical trial, a…
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