In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare conditions. A rare disease or condition is defined as…
Data from a Phase 2 trial examining BI 10115550, an oral treatment for idiopathic pulmonary fibrosis (IPF), has just been released. This data shows that this therapy is able to…
According to a story from the BHD Foundation, Patient Worthy partner The Myrovlytis Trust, along with the BHD Foundation and Pulse Infoframe, are collaborating on an international Birt-Hogg-Dubé (BHD) syndrome…
In the past, there have been a variety of research studies exploring and working to learn more about cystinuria, a rare genetic condition causing cystine, an amino acid, to accumulate…
Sometimes, it can be difficult to prompt the research of and creation of therapeutics for rare diseases. This is one of the reasons why the FDA created its Orphan Drug…
Every year, people from across the world come together to celebrate Myasthenia Gravis Awareness Month during the month of June. The Myasthenia Gravis Foundation of America (MGFA) plays a big…
In 2022, the 25th Annual Meeting of the American Society of Gene & Cell Therapy took place from May 16th-19th. During the meeting, researchers and other stakeholders discussed the latest…
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