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…
According to a story from the East Bay Times, it was 2003 when Isabel Bueso and her family traveled to the US from Guatemala. She was just eight years old,…
According to a recent article, researchers at the WORLDSymposium revealed they are looking into using pentosan polysulfate sodium to treat Maroteaux-Lamy syndrome. MPS VI (Maroteaux-Lamy Syndrome) Maroteaux-Lamy syndrome is…
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…
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…
Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the…
According to a story from Buzzfeed News, Maria Isabel Bueso was born in Guatemala with mucopolysaccharidosis VI, also known as Maroteaux-Lamy syndrome. At age seven she traveled to the US…
The Isaac Foundation The Isaac Foundation was established by a man named Andrew McFadyen. He created the organization in 2007 in honor of his son who is diagnosed with the rare…
Meet Ethan Ethan Faneran-Burley was diagnosed at two years old with Mucopolysaccharidosis type 6 (or MPS type 6). It's a progressive, degenerative, and ultimately fatal condition. Those with MPS type…
According to a story from the San Francisco Chronicle, Isabel Bueso's parents were told when she was born that she would not live more than seven years. However, she continued…
Yesterday, a group of experts presented "Changing lives: Long-term outcomes of MPS IVA & VI patients" at the Satellite Symposium sponsored by BioMarin. The program began with insights on the…
Life is a reflection of our attitude, and we can learn a thing or two from Alena Galan, a young woman who’s living the dream as a college freshman, hosting…
If you have Maroteaux Lamy syndrome, there's good news and not-so-good news. On the one hand, an understanding of this rare genetic disease has been reached by the medical community.…
Keenan Cahill is a YouTube sensation who just happens to have Maroteaux-Lamy syndrome. Although, after watching his channel for a half hour, he isn't letting it hold him back from…
Maroteaux-Lamy Syndrome, also known as mucopolysaccharidosis (MPSVI), is a lysosomal storage disorder. It's one of a number of disorders that affects specific enzymes in specific locations. The job of these…
I was reading an article in Rolling Stone magazine about a young internet celebrity, and buried at the end of the overblown descriptions of the young man's activities on Vine…
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