Pompe Disease Treatment Market Trends

Pompe Disease Treatment Market Trends

• The rising occurrence of Pompe disease, classified as a rare disorder, and the limited treatment options available, has prompted pharmaceutical and biotechnology companies to intensively focus on research. Their aim is to glean a deeper understanding of disease mechanisms and to innovate treatment avenues, including ERT, gene therapy, autophagy suppression, and combination therapy among others.
   
• The new approach of two-component therapy has gained popularity in the market, offering a promising opportunity for potential treatment options. Thus, the rising R&D initiatives concentrated on novel treatment approaches for rare diseases are anticipated to drive market growth in the coming years.
   

However, the treatment for Pompe disease often incurs substantial out-of-pocket expenses, thereby hindering patient access in low- and middle-income economies.