Written by Dawn Laney, MS, CDC, CCRC The journey to a Fabry disease diagnosis is rarely a clear, straight path. Fabry can mimic more common health issues and lead patients…
The FDA has recently granted ONCOFID®-P the Orphan Drug designation for the treatment of malignant mesothelioma (MM). This designation is reserved for therapies indicated for rare diseases, and it provides…
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…
For many patients, certain treatments can be expensive, invasive, or difficult to access. But it doesn't have to be this way. According to aHUS News, researchers are running two separate…
Orphan drug designation is granted in the United States to expedite the development and review of drugs and biologics intended to treat patients with rare conditions (over 200,000 Americans). Once…
For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…
As many people know, writing a book is a huge accomplishment - and one that many people wish to achieve throughout their lifetime. For Samantha VanAlstyne ("Sam"), a 25-year-old who initially…
A study found on Medscape confirms that the human immune system and the human brain are the most complicated in our biological systems. The two systems are responsible for…
Over ten years ago two Nobel-Prize-winning biologists, Emmanuelle Charpentier and Jennifer Doudna, discovered a technology called CRISPR (clustered regularly interspaced short palindromic repeats). Bacteria contain genetic sequences repeated throughout…
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