The document below was created by Dr. Albert Misko (pediatric neurologist, Massachusetts General Hospital) to generate improved care for the MLIV community. Since 2016, Dr. Misko has worked closely with the MLIV patient community and was specifically funded by a University of Pennsylvania grant through the Orphan Disease Center for natural history research to understand mucolipidosis type IV and MLIV patients in great detail. One of several outcomes from that research is this document.
The document offers:
- a description of the disease for anyone new to it (such as newly diagnosed families or doctors unfamiliar with MLIV);
- a list of care providers recommended that you assemble as your child's care team and what they should be monitoring, with possible suggestions;
- a list of the examinations and tests performed if a person with MLIV were to be seen at MGH so you can advocate for your child when s/he is seen elsewhere.
Families of ML4 patients are encouraged to download and print this document, and to share this with any medical provider you see. Please direct any questions about the document or suggestions to improve content or clarity to Rebecca Oberman.