Myrtelle Inc. has just announced that they have been granted three new designations from the FDA for their investigative gene therapy called rAAV-Olig001-ASPA. This therapy is being investigated as a…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Juvena Therapeutics has just announced that they've received a new grant which will fund their research of myotonic dystrophy type 1 (DM1). This was a very competitive grant which totals…
Awareness and advocacy are extremely important to every rare disease, and porphyria is no different. That's why we celebrate Porphyria Awareness Week (PAW) every year from April 2nd until the 9th.…
Currently, there are no cures for idiopathic pulmonary fibrosis (IPF), and the two available treatments aim to reduce lung scarring and symptom progression. However, it is clear that new therapeutic…
According to a recent article, Canadian actor Cameron Mathison opened up about dealing with Legg-Calve-Perthes disease throughout his childhood. Perthes Disease Perthes disease is a condition that affects the hips…
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