We continue to see strong commercial momentum and expansion into additional geographies. 8226;Pompe clinical program milestones. AT-GAA demonstrated a similar safety profile to alglucosidase alfa. The difference between the two groups was not statistically significant (p=0.60). There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. We may rely, in some circumstances, on trade secrets to protect our technology. Celenex has an exclusive license agreement with Nationwide Children's. This may have the effect of making branded products less attractive to buyers. The cost of preparing and submitting an NDA or BLA is substantial. The FDA may also undertake an audit of nonclinical and clinical trial sites. This is the start of the second phase of the procedure: the decision-making process. This is the case even when the studies\' results are negative. E.U. Exit") Regulations 2019 (the "Exit Regulations"). The Affordable Care Act has been subject to challenges in the courts. Our passion for making a difference unites us. We also believe that diversity of experience and thought is essential. In some cases, the FDA will approve gene therapies based on Phase 2 clinical trial data. Our competitors have also received orphan designation. Most of the state carry forwards generated prior to 2009 have expired through 2016. We are subject to the periodic reporting requirements of the Exchange Act. In addition, we may not pursue or obtain patent protection in all major markets. Additional funds may not be available on terms that are favorable to us, or at all.