Pioneering Genetic Testing Access Through Probably Genetic’s Whole Exome Sequencing for Alpha-Mannosidosis
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Pioneering Genetic Testing Access Through Probably Genetic’s Whole Exome Sequencing for Alpha-Mannosidosis

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…

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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit
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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit

  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…

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ISMRD International Conference for Glycoprotein Storage Diseases Coming This July

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…

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A New Treatment For Alpha-Mannosidosis Just Got Authorized By The EMA
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A New Treatment For Alpha-Mannosidosis Just Got Authorized By The EMA

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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