Gaucher Community Alliance

It’s officially time to register for our TEEN AND YOUNG ADULT ADVOCACY TRIP to Washington, DC! Registration, FAQs, and hotel booking all found on our website here: https://lnkd.in/eXawz6St

Join us virtually April 9th for our next educational webinar! Register here and read below for more details on our wonderful speakers. https://lnkd.in/een96_4F PRESENTERS: Prof. Ida Vanessa Doederlein Schwartz, MD Prof. Ida Vanessa Doederlein Schwartz, MD is a full professor in the Department of Genetics at the Universidade Federal do Rio Grande do Sul (UFRGS) in Brazil and the head of the Inborn Metabolic Diseases Clinics at the Hospital de Clínicas de Porto Alegre (HCPA), Brazil. She is also the coordinator of the Brazilian National Institute on Rare Diseases - InRaras. Currently, Dr. Schwartz is the President of the Brazilian Society of Medical Genetics and Genomics and the principal investigator of the Spur Therapeutics gene therapy trial for Gaucher disease. She has published over 200 indexed articles, with an H-index (Web of Science) of 37. Dr. Philip Yin, MD, PhD Dr. Philip Yin is a physician-scientist with a strong interest in innovative technologies to advance drug development, clinical care, and personalized medicine. With a background in internal medicine and infectious disease, Dr. Yin brings over 15 years of experience in the pharmaceutical industry, leading clinical development programs at both large pharmaceutical and smaller biotechnology companies. He serves as Head of Clinical Development at Spur Therapeutics, leading efforts to advance FLT201 gene therapy for individuals with Gaucher disease.

Join us at the Anne Kolb Nature Center in Hollywood, FL on April 27th for our next patient & family meeting! Lunch will be provided. Register here: https://lnkd.in/eNXbDtQY

Registration for the 2025 Spring Fling Music Fest is OFFICIALLY open! Join us in Riverside, IL for a fun, music-filled day on Saturday, April 26th. We are so honored to be apart of this event! Register below! https://lnkd.in/d2cqh4xe

Today is Rare Disease Day! Worldwide, there are more than 300 million people with a rare disease. Make sure to show your stripes, share your rare voice, and be an advocate for yourself today, and every other day. See below to send a letter to Congress! https://lnkd.in/eZGVaj-x

Rare Disease Week 2025 was an absolute success! Thank you EveryLife Foundation for Rare Diseases for hosting such an important and powerful event and giving us the tools to advocate for ourselves as rare disease patients, families, and caregivers. Click the link below to make a difference by signing a petition to Congress to continue their support of steady and robust federal agency leadership, funding, and resources. https://lnkd.in/eaPmKkZX

Join us this week on Capitol Hill with EveryLife Foundation for Rare Diseases for Rare Disease Week! We can’t wait to meet other rare disease advocates and make our voices heard by many members of Congress and government officials. See you there!

Join @TriageCancer for a FREE webinar about Taking Time Off Work on 3/27! Register: TriageCancer.org/Webinars. Your rights matter. This webinar will explain key federal laws like FMLA and ADA, and state and local leave laws, how these laws work together and other important health care rights at work. #TriageTalks #CancerRights #RightsatWork

Cyndi had the pleasure of attending the 2025 Sanofi Patient Advocacy Council in Washington, DC. The Council provides a unique platform to address issues impacting patient access and to recognize the importance of the patient voice. Cyndi represented GCA by sharing our 2025 priorities and goals for the year. Thank you, Sanofi!

Today marks IGT's 5th anniversary! Together, with a new administration and Congress, we remain committed to educating about the value of #genetherpy and the importance of accurately assessing its impact for patients, families and our healthcare system. Read more from IGT Chair Erik Paulsen - https://lnkd.in/eCqpS3GE