I had learned that the many twists and turns on our rare disease path were similar to the families we met Originally published on May 11, 2022, this article explores celebrates author…
"Rock Bottom" is the story of a family who’s second born child, a son named Ashton, was born with Mucolipidosis Type II, a disease so rare and so frequently mistaken…
During the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, which took place virtually from May 11-14, 2021, biotechnology company M6P Therapeutics ("M6P") presented preclinical data…
Upcoming Conference The International Society for Mannosidosis & Related Diseases (ISMRD) serves as an international patient advocate for those living with glycoprotein storage diseases. These include alpha-mannosidosis, fucosidosis, mucolipidosis type…
Researchers at the Massachusetts General Hospital Research Institute and the Georgia Institute of Technology are investigating a medication currently approved to treat a type of multiple sclerosis as a possible…
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