Fetal therapy offers hope for unborn patients suffering from the rare genetic disease Hunter Syndrome Some parents-to-be have to face the terrifying news that their unborn child has been diagnosed with a life-threatening genetic disorder.

Today, May 15, we proudly observe #MPSAwarenessDay and stand in solidarity with the #MPS community. We thank our patients who participated in the prenatal ERT #PEARLTrial. Their courage and their contributions are humbling. Meet one of these inspiring families: bit.ly/3OgxCZD #MPSAwarenessDay2025

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...

Attending #ACMGMtg25? Learn about the latest advances and clinical findings at our poster presentations and Learning Lounge on Thursday, March 20. We hope to connect with you in LA! #PEARLTrial @theacmg.bsky.social

Honoring #RareDiseaseDay and the strength of the #rare community. Your voices drive progress, and together, we’re working toward better treatments. #RareDiseaseAwareness #PEARLTrial pearltrial.ucsf.edu

Join us at #WORLDSymposium for interim results from the @ucsanfrancisco.bsky.social #PEARLTrial a first-in-human Phase 1 trial of In Utero #EnzymeReplacementTherapy for Lysosomal Disorders (PI Tippi MacKenzie). Presented by Billie Lianoglou: Thurs, Feb 6, 8:30 am #WORLDSymposia
pearltrial.ucsf.edu