Dear Pompe Patient Community Leaders,
At Audentes Therapeutics, we are focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases. As with many rare conditions, there is limited published information available about the humanistic impact, or burden, that the condition has on the individuals and families living with it every day. While there is published literature discussing the clinical and humanistic burden of Pompe disease, there are still gaps in fully understanding certain aspects of the humanistic burden, particularly for infantile-onset Pompe disease (IOPD).
We believe that understanding the perspective of those living with Pompe disease and how it affects them is important to developing our clinical plans for new therapies to treat Pompe disease. Therefore, we recently partnered with medical experts to conduct and publish a comprehensive review, entitled, “The Humanistic Burden of Pompe Disease: Are There Still Unmet Needs? A Systematic Review.” This article was published in the journal called BMC Neurology and can be found here.