Texas Infant to Become Fifth Child to Receive Life-Saving Gene Therapy Treatment on U.S. Soil DALLAS, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Sufyan Pashai, a 4-month-old from Dallas, will travel to Minnesota and become the fifth child to receive life-saving gene therapy treatment on U.S. soil for...

Inspirational Child with MLD, Calliope Joy, Passes Away BALA CYNWYD, Pa., March 25, 2022 (GLOBE NEWSWIRE) -- Calliope ‘Cal’ Joy Carr, 12, who inspired a global movement to raise awareness of and funding for rare diseases, passed away yesterday, Thursday,...