Comment from Kyrsten Lawless

See attachI am a mother to a child living with an ultra rare lysosomal storage disorder called Alpha Mannosidosis. Since diagnosis at age 5 our life has had to completely change. Rather than approving gene therapy specifically for Hurler's disease, MPS related disorders, etc can the same clinical trials look at approving gene therapy as a whole which then can be applied to different disease processes across the board? (In the case of lysosomal storage disorders) There are 50-70 different disorders that are considered lysosomal storage disorders. Alpha mannosidosis is not even listed when asking Google for an extensive list of all the diseases that are considered in the Lysosomal Storage Disorder Category. We are desperate for a gene therapy, and understand that there are risks. Please make it easier to develop therapies/ drugs for children affected by rare disease especially ones that are neurodegenerative. Lysosomal storage disorders are awful, and are caused by one missing/ messed up enzyme. We have: AI, Alphafold, AlphaGenome now that make gene therapies actually possible. Please encourage research for treatment of Lysosomal Storage Disorders as whole, Many are so rare that the sample size for a clinical trial would be too small. Enzyme replacement therapy does not cross the blood brain barrier. We also have to travel once a week for infusions. My daughter is 8 years old and doesn't understand what Alpha mannosidosis is, she doesn't understand why she has to get poked in the chest with a needle once a week. She has been doing this for 2.5 years now, and it is a battle every Monday. Some days she has to be restrained.ed file(s)