A recent article from Pharmaceutical Manufacturer highlights a significant achievement in the rare disease sector, as Rare Disease Biotech announced reaching a major milestone in the development of its new…
PTC Therapeutics has just announced that their therapy Waylivra (volanesorsen), a treatment for familial chylomicronemia syndrome (FCS), has been given Category 1 classification from the Drug Market Regulation Chamber in…
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…
According to a story from globenewswire.com, Akcea Therapeutics and Ionis Pharmaceuticals recently announced the release of top line results from a clinical study. This trial tested the drug WAYLIVRA as…
Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…
Familial partial lipodystrophy (FPL) is a rare genetic disorder that causes fat cells to die off. This makes people with this disorder suffer from the progressive loss of body fat. At…
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