Pompe mouse study

Adding miglustat to Lumizyme or Nexviazyme no help to mice

Adding the enzyme stabilizer miglustat did not improve the effectiveness of Lumizyme (alglucosidase alfa) or Nexviazyme (avalglucosidase alfa) in a mouse model of Pompe disease, a new study reports. “These data demonstrate that co-administration of miglustat with either [Lumizyme or Nexviazyme] to Pompe mice provides no additional…

Damage to muscle spindles may cause Pompe motor problems

Motor problems in people with Pompe disease may be explained by damage to muscle spindles, which are sensory receptors that detect muscle movement and help regulate muscle tone and maintain posture, a mouse study suggested. Muscle spindle structure and function were “severely compromised” in Pompe mice, resulting in a…