New insights from #WORLDSymposia 2026 are shaping how we think about #LysosomalDiseases. Our latest research update explores how early biomarker and symptom patterns in newborn‑screened infants may help guide #PompeDisease treatment decisions #MedSky
ow.ly/5X7Q50YgCLc
WORLDSymposium 2026 Original Article | Feb. 6, 2026 | NEJM.org AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial Fig. 1C. GM1 Ganglioside Level in Cerebrospinal Fluid The NEJM identity sits at the bottom.
Presented at #WORLDSymposia:
In nine patients with GM1 gangliosidosis, a fatal neurodegenerative disease, AAV9 β-galactosidase gene therapy was associated with short-lived adverse events, increased β-galactosidase levels, and delayed progression. Full trial results: nej.md/4rxOZpP
#MedSky
Contact me at lwuerth@rarelifesolutions.com to connect and learn how we can make a difference in the lysosomal disease community and explore innovative solutions in lysosomal disease publications and scientific communications.
#WORLDSymposia #LysosomalDisease #RareDiseaseAwareness #ForUsItsPersonal
Contact me at lwuerth@rarelifesolutions.com to connect and learn how we can make a difference in the lysosomal disease community and explore innovative solutions in lysosomal disease publications and scientific communications.
#WORLDSymposia #LysosomalDisease #RareDiseaseAwareness #ForUsItsPersonal
Contact me at lwuerth@rarelifesolutions.com to connect and learn how we can make a difference in the lysosomal disease community and explore innovative solutions in lysosomal disease publications and scientific communications.
#WORLDSymposia #LysosomalDisease #RareDiseaseAwareness #ForUsItsPersonal
Contact me at lwuerth@rarelifesolutions.com to connect and learn how we can make a difference in the lysosomal disease community and explore innovative solutions in lysosomal disease publications and scientific communications.
#WORLDSymposia #LysosomalDisease #RareDiseaseAwareness #ForUsItsPersonal
📹The CheckRare team spent the week at #WORLDSymposia 2025
Stay tuned for our interviews from the conference!
#CheckRare #RareDisease
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
