Mucolipidosis Archives - Patient Worthy

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How this Sanfilippo Syndrome Patient Registry is Helping Patients

Post author:Kendall Mason
Post published:May 1, 2020
Post category:MPS III (Sanfilippo Syndrome)Mucolipidosis Rare Disease

The Connect MPS Patient Registry was created in 2016 to learn more about Sanfilippo syndrome and mucolipidoses (ML). 24 advocacy organizations are a part of this registry, all of which…

Math Caujungco Receives Award for Mucolipidosis Type IV Discovery

Post author:Trudy Horsting
Post published:January 21, 2019
Post category:Dysautonomia Mucolipidosis

Mucolipidosis Type IV Mucolipidosis Type IV (ML-IV) is a rare disease which causes a reduced mental capacity and blindness. ML-IV is caused by a missing or malfunctioning TRPMLI protein. Most patients never…

How this Sanfilippo Syndrome Patient Registry is Helping Patients

Math Caujungco Receives Award for Mucolipidosis Type IV Discovery

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