Highlights of the Late-Onset Treatment Study (LOTS) – a compilation for Patients
New information regarding the use of enzyme replacement therapy (alglucosidase alfa) in children and adults with Pompe disease is now available following the completion of the Late-Onset Treatment Study (LOTS). LOTS was the first large-scale study that demonstrated the safety and efficacy (overall ability to produce a therapeutic effect) of alglucosidase alfa in children and adults with Pompe disease.