Bored Panda on MSN3 个月
This Family Advocates For Rare Sanfilippo Syndrome In Hopes That Others Have Better Chances
The average life expectancy is mid to late teens ... and children born with Sanfilippo syndrome will have a second chance at ...
Cenla 2-year-old becomes diagnosed with rare terminal disease
Two-year-old Lydia Rachal was diagnosed with Sanfilippo Syndrome Type B. The disease is terminal, and the life expectancy ...
Nasdaq1 个月
RARE Submits BLA to the FDA for Sanfilippo Syndrome Gene Therapy
Per Ultragenyx, approximately 3,000 to 5,000 patients in commercially accessible geographies are affected by this disease, with a median life expectancy ... III Angelman Syndrome Study for ...