About LOPD

LOPD is a degenerative muscle disease that can cause irreversible muscle damage even with management.1-3

This potentially irreversible decline in respiratory and motor function can occur in any patient with LOPD whether ERT-naive or ERT-experienced.3-6

DECLINE IN RESPIRATORY FUNCTION

Respiratory muscle weakness

Difficulty breathing and respiratory infections2

DECLINE IN MOTOR FUNCTION

Proximal muscle weakness

Difficulty ambulating, limited gross and fine motor skills2

A patient could decline at any time. Even with management, many patients declined after 3-5 years.

Patients typically experienced a period of benefit, followed by a plateau and decline. However, individual variances did occur.3,4,7,8

Improvement of respiratory function and walking distance is the goal of LOPD treatments

Considering that improvement or stabilization of respiratory function and walking distance is the goal of LOPD treatments, a change in therapy may be needed for patients with LOPD.2,5,9