Aro Biotherapeutics are developing a substrate reduction therapy for Pompe disease; here is a recording of the company’s detailed presentation about the Pompe therapy to another US patient group, Chelsea’s Hope. This group support Lafora disease and a similar approach may work well for them also.
Aro Biotherapeutics are a US company developing an alternative to ERT for Pompe disease; it’s a Substrate Reduction Therapy (SRT) that aims to reduce the glycogen stores in muscle cells that cause damage and loss of function. We hope to see clinical trials begin sometime next year.