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Stay up to date on our latest news about rare disorders
Leah Svarny, from LDRTC, presented at the 2nd IWGGD Scientific Symposium in Belgrade, Serbia.

LDRTC Symposium: Newborn Screening and Lysosomal Disorders
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Behind the Mystery: Dr. Ozlem Goker-Alpan Shares her Expertise on Managing People with Gaucher Disease
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Chiesi Global Rare Diseases Announces FDA Approval of ELFABRIO® for the Treatment of Fabry Disease
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First patient dosed in the GALILEO-1 study for Gaucher Disease
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Dr. Ozlem Goker-Alpan was nominated by patients and recognized as the Top LSD Specialist of 2023

LDRTC participates in studies that contributed to a new treatment option for late-onset Pompe disease

| April 7, 2024 | Gaucher Disease Patient & Family Workshop

| February 10, 2024 | FSIG Expert Fabry Conference

| February 8, 2024 | LDRTC's first Satellite Symposium at WORLDSymposium 2024

| October 15, 2023 | 2023 Gaucher Community Alliance Patient and Family Regional Meeting

| October 14, 2023 | LDRTC team is at Team Sanfilippo Fest IV in Pennsylvania

| September 28, 2023 | National MPS Society’s 37th Annual Family Conference in Maryland

Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease
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| September 21, 2023 | Our Team is at the 13th Annual Fabry Family Education Conference

| August 29, 2023 | Dr. Goker-Alpan at SSIEM 2023

| July 21, 2023 | 2023 NNPDF Family Support & Medical Conference

| May 6, 2023 | Gaucher Disease Patient & Family Education Meeting

LDRTC at WORLDSymposium™ 2023
LDRTC 's Poster Presentations
Basic Science – Poster Session I – February 22
Poster# 184 Margarita Ivanova - Gender differences in circulating inflammatory, immune, & tissue growth markers associated with Fabry disease-related cardiomyopathy Kiosk# 22-A
Poster# 196 Neil Kasaci Caspase inhibitors can counteract inflammasome activation and caspase-1 mediated fibrosis in Fabry disease Kiosk# 24-A
Translational Research – Poster Session III – February 23
Poster# 84 Julia Dao - TRAP5a and TRAP5b biomarkers in Gaucher disease Kiosk#8-A
Clinical Applications – Poster Session V – February 24
Poster# 138 Ozlem Goker-Alpan - The survival as relates to the clinical spectrum, molecular variants and chaperone response in acute neuronopathic Gaucher disease Kiosk#17-A

| August 5, 2022 | National MPS Society Conference in Nashville

| May 13, 2022 | Vaccine for Adolescents

| April 10, 2022 | FSIG Fabry Conference

| July 29, 2021 | Top 10% Most Cited Paper Published

| February 11, 2021 | Dr. Goker-Alpan Speaks at WORLDSymposium

| February 7-11, 2022 | WORLDSymposium
