“Today we have 56 members representing 54 countries around the globe and represent approximately 85% of the global Gaucher community” Tanya Collins-Histed, CEO of International Gaucher Alliance on the importance of international collaboration. bit.ly/InternationalG… #Lysosomal AVROBIO

We are excited to see significant movement in the stem cell & gene therapy trial! Three adults with cystinosis have been transplanted and all three are doing well and remain off cysteamine treatment! Thank you to the patients who volunteered for the trial! #cystinosis #hope

Now enrolling: The GuardOne study of an investigational gene therapy for #Gaucher disease type 1. To find out if you qualify and get more information on this study, call 1-877-330-5214 or visit bit.ly/3qQxEsR.

We’re excited to join @rareadvocates for Virtual Rare Disease Week 2021 on Capitol Hill, which brings together #raredisease community members to share their unique stories with legislators. This unique event is held July 14-22. Register here: rareadvocates.org/rdw #raredc2021

New research evaluated more than 5,800 foods and their impact on human health & the environment. An astonishing finding? Eating a hot dog could cost you 36 minutes of healthy life, & eating a serving of nuts instead could help you gain 26 minutes. READ: myumi.ch/E3MgX

In partnering with CoRDS, CRF has created the ONLY cystinosis patient registry in the world. Enrolling in the cystinosis registry is one of the best ways to help support research for those living with cystinosis. Join the fight against cystinosis: bit.ly/3fyBUL6

Thank you Rare Revolution for working on this series. Grateful to have the opportunity to share my hopes and dreams with the rare community. RARE Revolution Magazine

Now available: New resources on #GaucherDisease! Learn how #genetherapy aims to be a one-time treatment for Gaucher disease and may address symptoms caused by the effect the disease has on the brain. bit.ly/3gyFlRC

ASGCT's Clinical Trials Finder allows #raredisease patients and families to search more than 3,000 active and recruiting #genetherapy and #celltherapy trials around the world. Search by location, enrollment status, or treatment modality: bit.ly/3mtzk9q

Don’t miss Stephanie Cherqui, Ph.D., our collaborator at UCSD, and Cristina Baricordi, Ph.D., one of our senior scientists, at the Metabolic Disorder Summit today and tomorrow! Learn more at bit.ly/3jjKq0Z

In partnering with CoRDS, CRF has created the ONLY cystinosis patient registry in the world. Enrolling in the cystinosis patient registry is one of the best ways to help support research & advance treatments. Join the fight against cystinosis: bit.ly/3fyBUL6

What’s our CSO, Chris Mason, presenting today during AVROBIO’s Sponsor Symposium at the ESGCT virtual congress? Turn up the volume and find out. #ESGCT #LysosomalDisorders

We are thrilled to welcome Essra Ridha, MD, MRCP, FFPM, as our new chief medical officer. See details of her appointment in our release: bit.ly/2XMUsAZ

Nearly 1 in 5 Americans is an unpaid caregiver to family, friends or neighbors. This month, we recognize the dedication and hard work of #caregivers. #NationalFamilyCaregiversMonth Find caregiving resources here: bit.ly/2WSECnZ

Last month, we presented new safety data from our clinical trials in #FabryDisease and #GaucherDisease type 1 at the 28th Annual ESGCT Congress. #ICYMI, check out our press release for more details: bit.ly/3G1Emol

On this day exactly one year ago, Jacob Seachord became a cystinosis hero! On November 16, 2020, Jacob became patient #3 in Dr. Cherqui's stem cell & gene therapy trial. Thank you Jacob - & all the other clinical trial patients - for bringing our dream for a cure into reality!

Did you know that 70% of genetic rare diseases start in childhood? As we celebrate #WorldChildrensDay, we recognize the many children around the world living with rare diseases. If your child is one of them, share their story to raise awareness: cutt.ly/LTTPROI

We had the pleasure of meeting Allison, whose son lives with #MPSII, also known as #HunterSyndrome. She shared their journey to diagnosis, navigating clinical trials, and hopes for future treatments. Thank you for sharing your incredible story with us during #NFCMonth!

What's the difference between #genetherapy, #celltherapy, and #geneediting? Learn about these approaches as well as common challenges, risks, and benefits, on our Patient Education website. bit.ly/3K1yJsp

Equity means: ✔️ social opportunity, 🏫 non-discrimination in education and work, and 🏥 equitable access to health, social care, diagnosis and treatment. Join us on 28 February for a more equitable society! ➡️Check out our equity toolkit: cutt.ly/wIWqdSV