Casey Martin is well known as the golfer who sued the PGA in 2001 and won a landmark 7-2 Supreme Court decision. According to a report by ESPN, with the…
The FDA grants certain designations, such as the Orphan Drug (ODD) and Fast Track designations (FTD), to medications intended to treat rare and severe disorders. Recently, they granted both of…
According to an InBrief PDF from biotechnology and RNA-targeted therapeutics company Ionis Pharmaceuticals ("Ionis"), topline data from a Phase 2 clinical trial highlighted the benefits of cimdelirsen (IONIS-GHR-LRx) for patients…
“The patient network is incredibly important,” Lina Williamson, PhD, explained to me. You see, in her childhood, Lina experienced a number of seemingly unexplainable symptoms: severe and sometimes debilitating leg…
In a late October news release from pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus"), the company shared that it would be collaborating with the Loulou Foundation, a private UK nonprofit dedicated…
It is often difficult to estimate the prevalence of a rare disease within the United States. For one, it can be difficult to find all of the patients with a…
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