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.

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...

I love and cherish the wonderful work done in #IOPD and the preceding project (hopefully, also subsequent) - but there is a period in #ODP that just had the most annoying reports. You know the one, when all the figures and tables were in links.