ISMRD is very excited for Kathryn Lawless, who is the first Alpha-Mannosidosis patient in the United States to receive Lamzede enzyme replacement therapy. Here, Kathryn’s mother Kyrsten talks about their experience with Lamzede infusions.

 

Kathryn (age 5 will be 6 on May 30) has been receiving Enzyme Replacement Therapy (Lamzede) through infusion once a week for three weeks now. We decided to do ERT because we believe in the power of modern day genetics, and all of her doctors have recommended this treatment. Kathryn is receiving this treatment at Cleveland Clinic.  

 

Kathryn has been doing amazingly these past 3 weeks. The first day after her first infusion we noticed a difference in her behavior. She is not as tired, more agreeable, and seems to be processing and following directions better. She is also talking more, and has more spontaneous language throughout the day. She is being more successful at becoming more independent with tasks such as dressing/ undressing herself, opening containers, and organizing her things at home. She is getting around better and having less falls/ accidents/ getting hurt. She has met her physical therapy goals (outside of school), and her physical therapist says there is such a big positive difference in her behavior and performance during therapy sessions and she has to create new goals for her. So we are working on riding a bike now. She is getting consistent 100% on her behavior charts from school, and teachers and staff have called me wanting to share how much harder she is working at school, and how well she is doing. She is tolerating and enjoying activities after school, before treatments we were barely making it through the school day.

Kathryn’s port is a little more difficult than most to access, because she has more fat tissue. Our total travel time to/from treatment is 2.5 hours, with 3-4 hour infusion visit. Benadryl is a pre-med, and Kathryn doesn’t do well behaviorally with Benadryl and gets pretty restless after 2-3 hours of taking it.  She has gained weight even though she has been more active. (We have an upcoming appt with endocrinology).