Patient Worthy is excited to highlight Probably Genetic's free genetic testing program for alpha-mannosidosis. The intricate web of rare diseases often leaves individuals and their families searching for answers and…
Prior to February 2023, there were no FDA-approved treatment options for non-central nervous system manifestations of alpha-mannosidosis. But in mid-February 2023, Chiesi Global Rare Diseases shared via news release…
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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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
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…
According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called…
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