I was diagnosed with Pompe's disease for approx. a year ago after several years of struggle to find out what was wrong with me. From being a fulltime working mother of two, to barely being able to walk, has been extremely tough, and I'm not even close to acceptance. But, it does (hopefully) come with time. But, over time, more "flaws" in the form of less and less functioning muscles, more and more reduced breathing capacity and more pain and more aids also come. So acceptance never feels closer, rather the opposite. But, now that I open myself up to say, "I have Pompes" - may suggest that I have actually come a little further with this acceptance 😉 I try to take the days as they come and are extremely grateful for all the wonderful people I have in my life - you lift me up and make even the worst days worth everything ❤️
I would say that the most disabeling factor for me now is the extreme fatigue. This fatigue makes me bed bound most days. So my biggest wish is that this eventually will get better.
Linn, 35, Norway
According to DelveInsight, Pompe is a rare disease with the prevalent cases of 1 in 28,000 in the United States, and worldwide incidence cases are estimated to be 1:40,000.
ReplyDeleteThe US Food and Drug Administration (FDA) approved a Biologic License Application for the orphan drug Myozyme (alglucosidase alfa, recombinant human [rh] GAA) for the patients with Pompe disease.
Moreover, to accelerate Pompe pipeline, companies like Amicus Therapeutics and Actus therapeutics are involved in advancing the Pompe therapy market with their lead candidates like AT-GAA and ACTUS-101, respectively.
Source: Pompe Disease Market Insights, Epidemiology and Market Forecast-2028