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Message to the Patients


At M6PT, you are at the heart of everything we do. Our mission is to translate the M6PT innovation into next generation therapies for individuals affected by LSDs – to relieve suffering, to restore health, to increase hope, and to improve lives.

We strive to understand and document the natural course of each LSD and its implications for the life of each affected individual. Likewise, we want to stretch our imagination for how the natural course of these devastating illnesses could be amended with effective, safe, and optimized treatments. Our primary functions of discovering, developing, and delivering treatments quickly spill over into community involvement and support: by understanding your challenges and what it’s like to live with a rare, devastating disease like a lysosomal storage disorder, we can better serve and support you and the wider community. We aim to create educational materials that are user-friendly, timely, and helpful. To do this, we will work diligently with investigators, physicians, advocates, and industry partners.

At M6PT, defeating disease influences everything we do. We look forward to building and sustaining strong relationships with you and others in your disease community. Please feel free to email us at if you have questions or would like to connect.

With best regards,
Pawel Krysiak