Infusion reactions and antidrug antibodies can complicate enzyme replacement therapy in #FabryDisease.
New in #JIMD: Janssens et al. examine their occurrence and whether preventive measures can reduce the risk.
🔗 doi.org/10.1002/jimd...
New in #JIMD: Janssens et al. examine their occurrence and whether preventive measures can reduce the risk.
🔗 doi.org/10.1002/jimd...
August 26, 2026 at 11:44 AM
Infusion reactions and antidrug antibodies can complicate enzyme replacement therapy in #FabryDisease.
New in #JIMD: Janssens et al. examine their occurrence and whether preventive measures can reduce the risk.
🔗 doi.org/10.1002/jimd...
New in #JIMD: Janssens et al. examine their occurrence and whether preventive measures can reduce the risk.
🔗 doi.org/10.1002/jimd...
🚀 Zacks Small Cap Research - PLX: Elfabrio Estimates Up
🔎 Read the full update:
➡️https://theminingnews.com/zacks-small-cap-research-plx-elfabrio-estimates-up/
#Protalix #Protalix #ElfabrioP#PRX115r#PLXe#FabryDiseaset#GoutTreatmentu#OrphanDrug##PhaseIIr#RELEASETrialu#BRIGHTStudyP#EMAA#CHMP#EUApprov
🔎 Read the full update:
➡️https://theminingnews.com/zacks-small-cap-research-plx-elfabrio-estimates-up/
#Protalix #Protalix #ElfabrioP#PRX115r#PLXe#FabryDiseaset#GoutTreatmentu#OrphanDrug##PhaseIIr#RELEASETrialu#BRIGHTStudyP#EMAA#CHMP#EUApprov
August 14, 2026 at 2:46 AM
🚀 Zacks Small Cap Research - PLX: Elfabrio Estimates Up
🔎 Read the full update:
➡️https://theminingnews.com/zacks-small-cap-research-plx-elfabrio-estimates-up/
#Protalix #Protalix #ElfabrioP#PRX115r#PLXe#FabryDiseaset#GoutTreatmentu#OrphanDrug##PhaseIIr#RELEASETrialu#BRIGHTStudyP#EMAA#CHMP#EUApprov
🔎 Read the full update:
➡️https://theminingnews.com/zacks-small-cap-research-plx-elfabrio-estimates-up/
#Protalix #Protalix #ElfabrioP#PRX115r#PLXe#FabryDiseaset#GoutTreatmentu#OrphanDrug##PhaseIIr#RELEASETrialu#BRIGHTStudyP#EMAA#CHMP#EUApprov
Just a few weeks after filing for #bankruptcy protection, #SangamoTherapeutics has found #buyers for some of its key assets, including a #Fabrydisease therapy and its #genomicmedicine platforms.
pharmaphorum.com/news/lilly-p...
pharmaphorum.com/news/lilly-p...
Lilly, PTC snap up bankrupt Sangamo's main assets
Just a few weeks after filing for bankruptcy protection, Sangamo has found buyers for some of its key assets, raising up to $263.5m.
pharmaphorum.com
August 13, 2026 at 2:29 PM
Just a few weeks after filing for #bankruptcy protection, #SangamoTherapeutics has found #buyers for some of its key assets, including a #Fabrydisease therapy and its #genomicmedicine platforms.
pharmaphorum.com/news/lilly-p...
pharmaphorum.com/news/lilly-p...
#FabryDisease is often diagnosed late due to diverse symptoms and limited diagnostics.
This review shows how #Multiomics is uncovering #biomarkers and therapeutic targets to support earlier diagnosis and personalized treatment.
#OpenAccess: doi.org/10.1016/j.ge...
This review shows how #Multiomics is uncovering #biomarkers and therapeutic targets to support earlier diagnosis and personalized treatment.
#OpenAccess: doi.org/10.1016/j.ge...
July 7, 2026 at 3:54 PM
#FabryDisease is often diagnosed late due to diverse symptoms and limited diagnostics.
This review shows how #Multiomics is uncovering #biomarkers and therapeutic targets to support earlier diagnosis and personalized treatment.
#OpenAccess: doi.org/10.1016/j.ge...
This review shows how #Multiomics is uncovering #biomarkers and therapeutic targets to support earlier diagnosis and personalized treatment.
#OpenAccess: doi.org/10.1016/j.ge...
Female Fabry disease remains difficult to predict. Can combining α-galactosidase A activity, lysoGb3 and X-chromosome inactivation improve phenotype classification? This study suggests a tri-parametric approach may provide a clearer picture. #FabryDisease #RareDisease #LSD
doi.org/10.1002/jimd...
doi.org/10.1002/jimd...
July 1, 2026 at 8:13 AM
Female Fabry disease remains difficult to predict. Can combining α-galactosidase A activity, lysoGb3 and X-chromosome inactivation improve phenotype classification? This study suggests a tri-parametric approach may provide a clearer picture. #FabryDisease #RareDisease #LSD
doi.org/10.1002/jimd...
doi.org/10.1002/jimd...
Alexion vs Rare Disease Data Center - Cures Fabry
https://orphandrug.online/alexion-vs-rare-disease-data-center-cures-fabry/?utm_source=bluesky&utm_medium=social
#fabrydisease #alexionefficacydata #rarediseasetreatment
https://orphandrug.online/alexion-vs-rare-disease-data-center-cures-fabry/?utm_source=bluesky&utm_medium=social
#fabrydisease #alexionefficacydata #rarediseasetreatment
June 10, 2026 at 3:41 PM
Alexion vs Rare Disease Data Center - Cures Fabry
https://orphandrug.online/alexion-vs-rare-disease-data-center-cures-fabry/?utm_source=bluesky&utm_medium=social
#fabrydisease #alexionefficacydata #rarediseasetreatment
https://orphandrug.online/alexion-vs-rare-disease-data-center-cures-fabry/?utm_source=bluesky&utm_medium=social
#fabrydisease #alexionefficacydata #rarediseasetreatment
Researchers identified a plasma proteomic signature that can detect cardiac involvement in #FabryDisease with high accuracy. Study in the Journal of Molecular Medicine.
Read here: https://bit.ly/4tAvCxo
#RareDisease #MedSky #Cardiology
Read here: https://bit.ly/4tAvCxo
#RareDisease #MedSky #Cardiology
Researchers Identify 10-Protein Panel That Detects Early Cardiac Involvement in Fabry Disease
A plasma proteomic signature that identifies cardiac involvement in Fabry disease may enable earlier detection and risk stratification.
bit.ly
April 9, 2026 at 2:27 PM
Researchers identified a plasma proteomic signature that can detect cardiac involvement in #FabryDisease with high accuracy. Study in the Journal of Molecular Medicine.
Read here: https://bit.ly/4tAvCxo
#RareDisease #MedSky #Cardiology
Read here: https://bit.ly/4tAvCxo
#RareDisease #MedSky #Cardiology
Dabbing cannabis concentrate is a whole other level.
Effective, medical-grade effects (re: for my qol mgmt for #FabryDisease).
Psa, do not operate heavy machinery afterwards.
Effective, medical-grade effects (re: for my qol mgmt for #FabryDisease).
Psa, do not operate heavy machinery afterwards.
a picture of a smiling moon with red eyes and the words time for red eyes
ALT: a picture of a smiling moon with red eyes and the words time for red eyes
media.tenor.com
March 20, 2026 at 5:36 AM
Dabbing cannabis concentrate is a whole other level.
Effective, medical-grade effects (re: for my qol mgmt for #FabryDisease).
Psa, do not operate heavy machinery afterwards.
Effective, medical-grade effects (re: for my qol mgmt for #FabryDisease).
Psa, do not operate heavy machinery afterwards.
Lyso-Gb3 levels were elevated earlier than clinical symptoms in infants with LO FD, increased significantly in the first 2 years of life, and remained stable during follow-up. bit.ly/4ufQL18 #GIMO #Globotriaosylsphingosine #LysoGb3 #FabryDisease #LaterOnsetVariants
February 28, 2026 at 2:11 AM
Lyso-Gb3 levels were elevated earlier than clinical symptoms in infants with LO FD, increased significantly in the first 2 years of life, and remained stable during follow-up. bit.ly/4ufQL18 #GIMO #Globotriaosylsphingosine #LysoGb3 #FabryDisease #LaterOnsetVariants
In a recent meta-analysis published in the @jimd-editors.bsky.social, #GeneticTesting identified #FabryDisease in female patients at 4 times the rate of enzymatic protocols.
Read here: https://bit.ly/3MTCqq6
#RareDisease #MedSky
Read here: https://bit.ly/3MTCqq6
#RareDisease #MedSky
Fabry Disease Often Missed in Women, But Genetic Testing May Close Diagnostic Gap
Genetic testing detects 4 times as many Fabry disease cases as enzymatic methods in women, an under-recognized patient population.
bit.ly
February 19, 2026 at 10:13 PM
In a recent meta-analysis published in the @jimd-editors.bsky.social, #GeneticTesting identified #FabryDisease in female patients at 4 times the rate of enzymatic protocols.
Read here: https://bit.ly/3MTCqq6
#RareDisease #MedSky
Read here: https://bit.ly/3MTCqq6
#RareDisease #MedSky
Women with Anderson–Fabry disease are still being missed.
This meta-analysis shows genetic testing detects ~4× more cases than enzymatic tests in high-risk women — especially after stroke.
Normal enzymes ≠ no Fabry.
doi.org/10.1002/jimd...
#FabryDisease #RareDisease
This meta-analysis shows genetic testing detects ~4× more cases than enzymatic tests in high-risk women — especially after stroke.
Normal enzymes ≠ no Fabry.
doi.org/10.1002/jimd...
#FabryDisease #RareDisease
February 9, 2026 at 5:59 AM
Women with Anderson–Fabry disease are still being missed.
This meta-analysis shows genetic testing detects ~4× more cases than enzymatic tests in high-risk women — especially after stroke.
Normal enzymes ≠ no Fabry.
doi.org/10.1002/jimd...
#FabryDisease #RareDisease
This meta-analysis shows genetic testing detects ~4× more cases than enzymatic tests in high-risk women — especially after stroke.
Normal enzymes ≠ no Fabry.
doi.org/10.1002/jimd...
#FabryDisease #RareDisease
Improving intrafamily communication in #FabryDisease is key to #earlydiagnosis. Expert insights offer strategies to overcome knowledge, emotional, and systemic barriers for better outcomes. bit.ly/49FJHmg #GIMO #FamilyScreening #GeneticCounseling #CommunicationBarriers
January 28, 2026 at 10:32 PM
Improving intrafamily communication in #FabryDisease is key to #earlydiagnosis. Expert insights offer strategies to overcome knowledge, emotional, and systemic barriers for better outcomes. bit.ly/49FJHmg #GIMO #FamilyScreening #GeneticCounseling #CommunicationBarriers
Isaralgagene civaparvovec (ST-920), Sangamo Therapeuticss’ investigational gene therapy for #FabryDisease, moved closer to potential approval after the US FDA accepted the company’s request for a rolling submission of its Biologics License Application (#BLA).
Read more: https://bit.ly/3Z11tKe
Read more: https://bit.ly/3Z11tKe
Sangamo Begins FDA Rolling BLA Submission of Isaralgagene Civaparvovec for Fabry Disease
Sangamo initiated a rolling BLA submission to the FDA for isaralgagene civaparvovec (ST-920), a gene therapy for Fabry disease.
bit.ly
January 8, 2026 at 7:03 PM
Isaralgagene civaparvovec (ST-920), Sangamo Therapeuticss’ investigational gene therapy for #FabryDisease, moved closer to potential approval after the US FDA accepted the company’s request for a rolling submission of its Biologics License Application (#BLA).
Read more: https://bit.ly/3Z11tKe
Read more: https://bit.ly/3Z11tKe
🧬 Fabry Disease Market expands with rising enzyme replacement therapies. Report: www.marketresearchfuture.com/reports/fabr... #FabryDisease #RareDiseases
Fabry Disease Market Size, Share, Trends, Report 2035
Fabry Disease Market growth is projected to reach 5.58 USD billion, at a 7.82% CAGR by driving industry size, share, top company analysis, segments research, trends and forecast report 2025 to 2035.
www.marketresearchfuture.com
December 10, 2025 at 12:49 PM
🧬 Fabry Disease Market expands with rising enzyme replacement therapies. Report: www.marketresearchfuture.com/reports/fabr... #FabryDisease #RareDiseases
Join us tomorrow for ‘#FabryDisease 2025: Advances in Diagnosis & Treatment for #Anderson-Fabry’ - a virtual conference on December 5, 2025 with 5.50 ACCME credits. Click for info: www.eventleaf.com/e/Fabry2025
#Kidney #Cardiology #Gastroenterology #ENT #Opthalmology
#Kidney #Cardiology #Gastroenterology #ENT #Opthalmology
December 4, 2025 at 3:41 PM
Join us tomorrow for ‘#FabryDisease 2025: Advances in Diagnosis & Treatment for #Anderson-Fabry’ - a virtual conference on December 5, 2025 with 5.50 ACCME credits. Click for info: www.eventleaf.com/e/Fabry2025
#Kidney #Cardiology #Gastroenterology #ENT #Opthalmology
#Kidney #Cardiology #Gastroenterology #ENT #Opthalmology
This recent review examines the #Pathophysiology of #Fabrydisease (FD) in its primary target organs and summarizes #Omics-based advances that hold great promise for tailored #therapeutic strategies in FD. #medsky
#OpenAccess: www.sciencedirect.com/science/arti...
#OpenAccess: www.sciencedirect.com/science/arti...
December 2, 2025 at 12:31 PM
This recent review examines the #Pathophysiology of #Fabrydisease (FD) in its primary target organs and summarizes #Omics-based advances that hold great promise for tailored #therapeutic strategies in FD. #medsky
#OpenAccess: www.sciencedirect.com/science/arti...
#OpenAccess: www.sciencedirect.com/science/arti...
#ASHG2025: Roberto Araujo, MD, PhD, Senior North America Medical Director for #Fabry and #Pompe disease at Sanofi, speaking Oct. 15 at the @geneticssociety.bsky.social 2025 conference in Boston.
#RareDisease #FabryDisease #PompeDisease #Sanofi #MedSky #RareSky
#RareDisease #FabryDisease #PompeDisease #Sanofi #MedSky #RareSky
October 16, 2025 at 3:08 PM
#ASHG2025: Roberto Araujo, MD, PhD, Senior North America Medical Director for #Fabry and #Pompe disease at Sanofi, speaking Oct. 15 at the @geneticssociety.bsky.social 2025 conference in Boston.
#RareDisease #FabryDisease #PompeDisease #Sanofi #MedSky #RareSky
#RareDisease #FabryDisease #PompeDisease #Sanofi #MedSky #RareSky
Sex differences in alpha galactosidase protein processing and its impact on disease severity in Fabry disease
Hughes, D., Kurdi, H. et al.
Paper
Details
#FabryDisease #AlphaGalactosidase #SexDifferencesInDiseaseSeverity
Hughes, D., Kurdi, H. et al.
Paper
Details
#FabryDisease #AlphaGalactosidase #SexDifferencesInDiseaseSeverity
October 10, 2025 at 4:03 PM
Sex differences in alpha galactosidase protein processing and its impact on disease severity in Fabry disease
Hughes, D., Kurdi, H. et al.
Paper
Details
#FabryDisease #AlphaGalactosidase #SexDifferencesInDiseaseSeverity
Hughes, D., Kurdi, H. et al.
Paper
Details
#FabryDisease #AlphaGalactosidase #SexDifferencesInDiseaseSeverity
#FabryDisease is severe and causes considerable burden in female patients, according to a study that appeared in the Journal of Medical Genetics. The study also showed that #Migalastat is efficient in the long term in these patients.
Learn more: https://bit.ly/3KB4ahQ
#RareDisease #MedSky #RareSky
Learn more: https://bit.ly/3KB4ahQ
#RareDisease #MedSky #RareSky
Migalastat Efficient in Female Patients With Fabry Disease
Fabry disease is severe and causes considerable burden in female patients and migalastat is efficient in the long term in these patients.
bit.ly
October 6, 2025 at 5:06 PM
#FabryDisease is severe and causes considerable burden in female patients, according to a study that appeared in the Journal of Medical Genetics. The study also showed that #Migalastat is efficient in the long term in these patients.
Learn more: https://bit.ly/3KB4ahQ
#RareDisease #MedSky #RareSky
Learn more: https://bit.ly/3KB4ahQ
#RareDisease #MedSky #RareSky
Sabina went from caring for her dad with Fabry disease to joining a clinical trial, and now she helps researchers shape studies around real family needs. See how patients are changing rare-disease research: www.clinicaltrialsforall.org
#RareDisease #FabryDisease #PatientVoice
#RareDisease #FabryDisease #PatientVoice
July 24, 2025 at 7:04 PM
Sabina went from caring for her dad with Fabry disease to joining a clinical trial, and now she helps researchers shape studies around real family needs. See how patients are changing rare-disease research: www.clinicaltrialsforall.org
#RareDisease #FabryDisease #PatientVoice
#RareDisease #FabryDisease #PatientVoice
A #genetherapy for #Fabrydisease could be on the horizon, after a clinical trial readout for #SangamoTherapeutics' ST-920 hit its safety and efficacy endpoints.
buff.ly/A51kdwd
buff.ly/A51kdwd
June 25, 2025 at 12:00 PM
A #genetherapy for #Fabrydisease could be on the horizon, after a clinical trial readout for #SangamoTherapeutics' ST-920 hit its safety and efficacy endpoints.
buff.ly/A51kdwd
buff.ly/A51kdwd
Many congratulations to our very own Magdalena Schuettler on her amazing Masters thesis defence on #FabryDisease with proud PI @iimog.sigmoid.social.ap.brid.gy
June 11, 2025 at 10:32 AM
Many congratulations to our very own Magdalena Schuettler on her amazing Masters thesis defence on #FabryDisease with proud PI @iimog.sigmoid.social.ap.brid.gy
CME: Fabry Disease Research Highlights
Learn more at checkrare.com/learning/p-f...
#CheckRare #CME #FabryDisease
Learn more at checkrare.com/learning/p-f...
#CheckRare #CME #FabryDisease
May 23, 2025 at 3:37 PM
CME: Fabry Disease Research Highlights
Learn more at checkrare.com/learning/p-f...
#CheckRare #CME #FabryDisease
Learn more at checkrare.com/learning/p-f...
#CheckRare #CME #FabryDisease
FYI: LucidQuest Views >>> Cell and Gene Therapy Weekly News – May 8th 2025 #News #FabryDisease #CARTTherapy #Glioblastoma #DMD Comment below!
Cell and Gene Therapy Weekly News – May 8th 2025
This Week in Gene & Cell Therapy: Gene Therapy Milestones, Strategic Shifts, and Breakthroughs in Oncology and Cardiology 🧬 From […]
The post Cell and Gene Therapy Weekly News – May 8th 2025 appeared first on LucidQuest Ventures.
dlvr.it
May 16, 2025 at 3:01 PM
FYI: LucidQuest Views >>> Cell and Gene Therapy Weekly News – May 8th 2025 #News #FabryDisease #CARTTherapy #Glioblastoma #DMD Comment below!
ICYMI: LucidQuest Views >>> Cell and Gene Therapy Weekly News – May 8th 2025 #News #FabryDisease #CARTTherapy #Glioblastoma #DMD Comment below!
Cell and Gene Therapy Weekly News – May 8th 2025
This Week in Gene & Cell Therapy: Gene Therapy Milestones, Strategic Shifts, and Breakthroughs in Oncology and Cardiology 🧬 From […]
The post Cell and Gene Therapy Weekly News – May 8th 2025 appeared first on LucidQuest Ventures.
dlvr.it
May 9, 2025 at 2:59 PM
ICYMI: LucidQuest Views >>> Cell and Gene Therapy Weekly News – May 8th 2025 #News #FabryDisease #CARTTherapy #Glioblastoma #DMD Comment below!