Gene therapy halts progression of rare genetic condition in young boy
Updated: 2024-06-28 15:38:49
Skip to content Menu Follow Us On Substack Google News Twitter X FaceBook Bluesky Our Bloggers Search Gene therapy halts progression of rare genetic condition in young boy June 28, 2024 The Hospital for Sick Children SickKids Michael is checked by a nurse at SickKids before receiving gene therapy in a single-patient clinical trial for spastic paraplegia type 50 SPG50 When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for Sick Children SickKids  in March 2022, the course of his condition was dramatically . altered Michael has spastic paraplegia type 50 SPG50 an âultra-rareâ progressive neurodegenerative disorder that causes developmental delays , speech impairment , seizures , a progressive paralysis of all four limbs ,