M6P Therapeutics

Making Pompe A Thing Of The Past

About this Event

Pompe disease is a rare, autosomal recessive, inherited disorder in which toxic levels of the complex sugar glycogen accumulates in cells. Pompe disease is estimated to affect less than one in 50,000 individuals worldwide. There are two forms of the disease:

Early Onset Pompe attacks babies who appear normal at birth, but exhibit symptoms by 2-3 months of age (or earlier). Progression is rapid. These infants are so severely affected that they become “limp” unable to feed or move. Their hearts become massively enlarged, and they typically die of cardio-respiratory failure before reaching 12 months of age.

Delayed Onset Pompe can present at any age. Glycogen build up is not as rapid as in the infantile form but the disease is progressive and can greatly decrease the life span of the afflicted person. Respiratory complications are the main cause of death. Delayed onset patients that present symptoms early in life are usually more severely affected and rarely survive past the second or third decade of life.

M6P is currently developing gene therapy products and enzyme replacement therapy options to treat individuals with Pompe disease and has received Rare Pediatric Drug Designation for their program.

Register now to watch this Fireside Chat with Tiffany House: President, AMDA-Pompe Association, and Cuong Do, Founder and Chairman of M6P Therapeutics to for the discussion of this devastating disease and the path to a cure.

Video On Demand

– Recorded

October 23

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