The passage of the Orphan Drug Act was an important step in creating an environment that supports rare disease research and drug development. This Act created Orphan Drug designation,…
The FDA has carried on a year-long disagreement with BrainStorm Cell Therapies, the developer of the experimental stem cell treatment called NurOwn. It is referred to as a personalized cell…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Theravance Biopharma’s late-stage failure suddenly became a jump-off point for treating multiple system atrophy (MSA) according to a recently published article in Neurology Live. After the failure of two…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Currently, the standards-of-care for narcolepsy include lifestyle changes involving diet and exercise, behavioral therapy, nap therapy, and certain medications designed to combat excessive daytime sleepiness. However, a majority of…
Data from the first cohort of the Phase 2 KEYNOTE-158 trial, evaluating pembrolizumab for individuals with advanced thyroid carcinoma, showed positive safety and efficacy. According to Cancer Network, 103 participants…
Drug development within the rare disease space can be tricky. In many cases, there is a general lack of research and understanding around these conditions; as a result, many…
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