Kyleigh Kista, a 3 year old from Benonin South Africa needs heart surgery within the next 3 months to save her life. Kyleigh was diagnosed with tetralogy of Fallot with…
The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this designation, "rare" conditions are those affecting…
According to a recent article, the preliminary results of a study suggest that certain oral and infusion therapies may be beneficial for those suffering from fatigue due to myalgic…
Life is filled with endless ups and downs. The first 5 of my ongoing 20 years living with dystonia were pretty much nothing but down times. I experienced the most horrible pain…
Pursuing an education can be difficult enough without any other extraneous factors. But what if you were also battling frequent infections, gastrointestinal problems, and recurrent hospitalizations? For Evan Brandon,…
X4 Pharmaceuticals Inc. has recently published data from 3 different posters which were presented at the American Society of Hematology (ASH) Annual Meeting in Georgia. You can read the full…
Prometheus Biosciences Inc. has just announced positive results from their Phase 1 investigation for a new treatment for systemic sclerosis-associated interstitial lung disease (SSC-ILD). This trial investigated PRA023, an anti-TL1A…
Back in December, UCB released the results of its phase 3 trial of rozanolixizumab in adult patients with generalized myasthenia gravis (gMG). Fortunately, these data were positive, pointing to this…
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