April 15 is #InternationalPompeDay. We stand w/the #Pompe community & are grateful to have been able to enroll 3 #IOPD patients in our prenatal ERT #PEARLTrial. We're excited by the positive impact, as seen in the story of the first family receiving the protocol www.nbcbayarea.com/news/local/b...
After Losing 2 Children to Rare Genetic Disease, Family Celebrates Healthy Daughter Thanks to Therapy Pioneered by UCSF Doctor
In a first-of-its-kind fetal therapy, Dr. Tippi MacKenzie, a pediatric surgeon at UCSF, began treating Ayla Bashir’s Pompe disease before she was even born. Garvin Thomas reports.
www.nbcbayarea.com
April 15, 2025 at 2:53 PM
April 15 is #InternationalPompeDay. We stand w/the #Pompe community & are grateful to have been able to enroll 3 #IOPD patients in our prenatal ERT #PEARLTrial. We're excited by the positive impact, as seen in the story of the first family receiving the protocol www.nbcbayarea.com/news/local/b...
This #InternationalPompeDay, head over to our YouTube channel to watch Professor Benedikt Schoser discuss the importance of looking beyond the core phenotype of #LOPD and understanding the associated psychological and emotional burden #MedSky #medicaleducation #PompeDisease
ow.ly/g7bT50VAGPE
ow.ly/g7bT50VAGPE
April 15, 2025 at 10:03 AM
This #InternationalPompeDay, head over to our YouTube channel to watch Professor Benedikt Schoser discuss the importance of looking beyond the core phenotype of #LOPD and understanding the associated psychological and emotional burden #MedSky #medicaleducation #PompeDisease
ow.ly/g7bT50VAGPE
ow.ly/g7bT50VAGPE