An opinion expressed by Sheila Frame, President of Americas at Amryt Pharma, was published recently in the Pittsburgh Post-Gazette. Ms. Frame, who serves on the board of the Rare…
Neuromyelitis optica spectrum disorder (NMOSD) can have long-term impacts for those affected. Current research suggests that up to 69% of patients may have severe unilateral or bilateral vision loss…
Enzyme replacement therapy (ERT) has been explored as a potential therapeutic option for mucopolysaccharidosis type III A (MPS III-A or Sanfilippo syndrome type A). However, these solutions have not…
What would you do if you were told that your child had a rare disease? How about if your child had an ultra-rare disease, which affects fewer than 20 in…
The other day I read a post in a dystonia support group that is similar to something I see and hear very often. I’m sure you have as well, and…
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…
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