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Mucolipidosis

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ASGCT: Proof-of-Concept Data Shared on M002 for ML II
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ASGCT: Proof-of-Concept Data Shared on M002 for ML II

  • Post author:Jessica Lynn
  • Post published:May 31, 2021
  • Post category:Mucolipidoses Type II/Mucolipidosis

  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…

Continue Reading ASGCT: Proof-of-Concept Data Shared on M002 for ML II
How this Sanfilippo Syndrome Patient Registry is Helping Patients
source: pixabay.com

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…

Continue Reading How this Sanfilippo Syndrome Patient Registry is Helping Patients
Math Caujungco Receives Award for Mucolipidosis Type IV Discovery

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…

Continue Reading Math Caujungco Receives Award for Mucolipidosis Type IV Discovery

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