Mark Tarnopolsky, MD, discusses how doctors can help people with Pompe disease evaluate new therapies, access treatment options, and measure whether a treatment is working.
Transcript
Yeah, I think for patients, they’re always asking about: “Are there new therapies that are available? Are they superior to what I have?”
And generally, good knowledge of the studies and good knowledge of the patient are critical for the doctor to help them provide, you know, knowledgeable information about what treatments are available, when and if to abandon a current treatment path and move to a new treatment path if that’s required.
And at that point, if there are options available, which now there are, how could they access it? Can they access that in their country? How do they go about accessing that?
And how do we go about measuring the efficacy of potentially new treatment? Are things that we need to discuss with the patient.
