Our team is composed of scientific experts and pioneers in #GeneTherapy who work with a sense of urgency to optimize an approach tailored to the unique features of each disease. We’re in pursuit of long-lasting, transformative treatments. More here: https://t.co/YtWrvKjsub

This #WorldHealthDay, we are recognizing the push towards a #HealthierTomorrow for ourselves and our planet. At Sio, our focus is to advance the treatment landscape for rare, fatal pediatric disorders. Learn more:  https://t.co/bltesvuxKP  @WHO

Today is #NationalDoctorsDay! We’d like to thank all the hardworking physicians around the globe for helping to care for and guide patients and families as they navigate the numerous challenges that come along with rare diseases.

Our mission is to combine cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients with debilitating #NeurodegenerativeDiseases. Learn more: https://t.co/jeD10PW4ZH

At Sio, patients and families are our priority. Visit our website to find educational resources for our #GeneTherapy treatments in development: https://t.co/KnvbIWHcs6

Meet our January MVP, Rob Wiley, Director, Project Management. Rob continues to exemplify our core value of moving quickly & making everyday matter. Rob moves with speed and purpose & has been invaluable in managing, problem-solving, and advancing our GM1 and GM2 programs.

#RareDisease patients wait an average of 6.3 years after symptoms present before receiving a confirmed diagnosis. Sio sponsors the Invitae Detect LSD program, which provides no-charge genetic testing & counseling for children suspected of having an LSD: https://t.co/lLpCyq0x26

Sio’s Chief Commercial Officer @MeswaniP spoke with @Inside_PM about increased investments in the #RareDisease space. Read the full article, including his thoughts on the value of patient organizations and the impact of the COVID-19 pandemic, here:

Today is #RareDiseaseDay! Rare diseases affect 300 million people globally. The Sio team is dedicated to changing the current treatment landscape of rare genetic diseases. Learn more: https://t.co/wtWuA7Ch7X #ShareYourClolors

Thank you to all the #RareDisease advocates out there, for your hard work and dedication in raising awareness and providing support for patients and their families. We are forever grateful. @CureGM1 @TaySachs @Tay_Sachs @_CTSF_

#DYK that newborn screening is performed in all 50 states? We have come a long way since The Newborn Screening Act started in the 1960s, but we still have a ways to go. In the US, only a few types of #RareDiseases are tracked when a person is diagnosed: https://t.co/o1LcpsCZI2

#RareDisease diagnosis can be difficult & patient populations can be relatively small, causing them to be overlooked by the research community. But at Sio, we are proud to fight for the #RareDisease patients and sponsor the Invitae Detect LSD program: https://t.co/azXj7qJ0Ah

#TaySachs and #Sandhoff diseases are the two distinct types of #GM2 gangliosidosis. This #RareDiseaseDay, we are raising awareness for this genetic disorder that has no approved treatments. Learn more here: https://t.co/MWUZOzJl9S

Today’s #RareDisease fact: Of the 4MM babies screened for serious disorders that are present at birth each year, screening identifies over 12K infants annually with a condition that, if left undiagnosed & untreated, would cause severe disability or death: https://t.co/WReBGnFeEO

Meet our December MVPs: Maggie Curran, Becky Kitchener, Hayde Eng, and Blake Griffin. This team was nominated for embodying our core value of moving quickly with their exceptional cross-functional collaboration to operationalize our new lab in North Carolina. Thank you all!

Today we announced corporate updates and fiscal third quarter 2021 financial results. We look forward to continuing to advocate for and working towards treatments for rare fatal pediatric diseases. Read the full release here: https://t.co/qGSzJjEDa8 $SIOX

In honor of #RareDiseaseDay this month, we're sharing #RareDisease facts all month long! There are many different causes of #RareDiseases. The majority of rare diseases are genetic, caused by changes in genes or chromosomes. Learn more: https://t.co/h5kh8Pe1nE

Answer: #GM1 gangliosidosis is an inherited lysosomal storage disorder (LSD) that progressively destroys nerve cells (neurons) in the brain and spinal cord. Thanks for participating! To learn more, visit: https://t.co/DUyBIn3nSC #RareDiseaseDay

We are proud to present at the 18th annual #WORLDSymposia 2022 this week. If you’re attending, check out our oral presentation on AXO-AAV-GM1 by Cynthia Tifft, MD, PhD of @genome_gov as well as two poster presentations. Visit the WORLDSymposium website: https://t.co/SMuyxGnTxM

Time for a #poll in preparation for #RareDiseaseDay2022! Fill in the blanks: #GM1 gangliosidosis is an inherited lysosomal storage disorder (LSD) that progressively destroys nerve cells (neurons) in the ______ and _______.