According to a press release from Ikena Oncology, the FDA has recently accepted an Investigational New Drug (IND) application for Ikena's investigational drug candidate, IK-930. This TEAD inhibitor is intended…
Written by Rod Cisneros Like many people, I keep a daily journal. I use it to document my everyday thoughts and I even include advice for family members who might…
In the European Union (EU), Orphan Drug designation ("Orphan designation") is granted to products intended to treat, prevent, or diagnose a life-threatening, chronic, or rare disease. These are defined as…
A recent article in NewScientist describes Borrelia burgdorferi as a bacterium that is the cause of Lyme disease (borreliosis). The trail of infection begins with wild mice that harbor the…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Canavan disease is an ultra-rare condition that currently has no FDA-approved therapies, meaning that patients face a severe unmet medical need. BridgeBio Pharma aims to change this with their investigational…
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