GM1 Gangliosidosis Affects Children Throughout the World

GM1 Gangliosidosis is not specific to a particular race, region, class, gender, or age group.  While GM1 is considered a rare disease, it’s prevalence is likely underestimated.  Some children may never properly be diagnosed, tragically perishing before the age of 2 in some infantile cases.

Furthermore, the average age of diagnosis for Juvenile cases is 11 years old.  Many of the children diagnosed with Juvenile may have endured a diagnostic odyssey or been misdiagnosed for many years.  Worse, these misdiagnosed children may not have received proper medical care as a result.

Type 3 or Adult Onset GM1 is extremely rare, the rarest of the rare.  Type 3 is believed to be most common in Japan.   These cases are commonly misdiagnosed as well.  Sometimes GM1 in adults may be mistaken for Parkinson’s disease.

As the field of medical genetics continues to advance, proper diagnoses will hopefully become more prevalent.  Hopefully, gene therapies will also advance.  It is our mission to ensure GM1 will someday be a disease of the past.  For now, the fight is on!



GM1 Gangliosidosis Affects Children Throughout the World
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