Fabry Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2024-08-20Clinical Trial Update: Screening of Fabry Disease in Patients With Left Ventricular Hypertrophy Detected in Echocardiography
The study completed screening of patients with left ventricular hypertrophy detected by echocardiography for Fabry disease. It assessed the …
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CLINICAL_TRIALS trial_result · 2024-08-09Clinical Trial Update: Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease [Phase 3]
Phase 3 trial evaluating oral lucerastat as monotherapy in adults with Fabry disease; results assessing efficacy and safety have been posted…
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CLINICAL_TRIALS trial_result · 2024-07-31Clinical Trial Update: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Open-label expanded access treatment with pegunigalsidase alfa for Fabry disease is described, providing therapy to patients outside of a tr…
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CLINICAL_TRIALS research · 2024-07-30Clinical Trial Update: Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease [Phase 2]
Clinical trial update for a Phase 2 study of AL01211 in treatment-naive males with classic Fabry disease; the study is currently recruiting …
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CLINICAL_TRIALS research · 2024-07-29Clinical Trial Update: Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease [Phase 3]
ISU Abxis Co., Ltd. is conducting a Phase 3 trial to evaluate the safety and efficacy of Fabagal (agalsidase beta) for Fabry disease, with p…
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CLINICAL_TRIALS research · 2024-07-22Clinical Trial Update: Molecular Imaging in Fabry Disease of the Heart
A University of Cambridge-led clinical trial is recruiting to study molecular imaging in Fabry disease of the heart (cardiac variant).
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FDA approval · 2024-07-10FDA Approved: FABRAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for FABRAZYME (agalsidase beta) by Genzyme Corporation.
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CLINICAL_TRIALS research · 2024-07-09Clinical Trial Update: Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease [EARLY_Phase 1]
A Phase 1 pediatric trial is evaluating a gene therapy drug for Fabry disease, focusing on safety, tolerability, and efficacy. The study is …
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CLINICAL_TRIALS trial_result · 2024-06-17Clinical Trial Update: A Study of Replagal in Treatment-naïve Adults With Fabry Disease [Phase 3]
This clinical trial update reports that the Phase 3 study of Replagal (agalsidase alfa) in treatment-naïve adults with Fabry disease was ter…
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CLINICAL_TRIALS research · 2024-06-12Clinical Trial Update: Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score [NA]
This clinical trial update examines the prevalence of Fabry disease in high-risk patients with left ventricular hypertrophy and the implemen…
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CLINICAL_TRIALS research · 2024-06-11Clinical Trial Update: Evaluation of a Screening Strategy of Fabry Disease in Patient With Renal Biopsy [NA]
Clinical trial evaluating a screening strategy for Fabry disease in patients undergoing renal biopsy; recruitment is ongoing at University H…
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FDA approval · 2024-06-05FDA Approved: GALAFOLD — Supplemental Application (Amicus Therapeutics US, LLC)
The FDA approved a supplemental application for GALAFOLD (migalastat hydrochloride) from Amicus Therapeutics US, LLC. The action updates the…
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CLINICAL_TRIALS research · 2024-05-23Clinical Trial Update: Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
A completed clinical trial update evaluating treatment-related benefits and patient satisfaction in Fabry disease. It provides insights into…
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CLINICAL_TRIALS trial_result · 2024-05-23Clinical Trial Update: French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat
A completed French prospective observational cohort study evaluating outcomes in Fabry disease patients treated with migalastat. Sponsor: Am…
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CLINICAL_TRIALS trial_result · 2024-04-10Clinical Trial Update: Study of the Effects of Fabrazyme Treatment on Lactation and Infants [Phase 4]
Phase 4 trial update on Fabrazyme (agalsidase beta) evaluating effects on lactation and infants in Fabry disease, with terminated status and…
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CLINICAL_TRIALS trial_result · 2024-04-08Clinical Trial Update: An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease [Phase 1, Phase 2]
Open-label Phase 1/2 trial exploring gene therapy 4D-310 in adults with Fabry disease.
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CLINICAL_TRIALS trial_result · 2024-03-15Clinical Trial Update: PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion [Phase 1]
Phase 1 pharmacokinetic/pharmacodynamic study comparing two agalsidase formulations given as a single 1 mg/kg intravenous dose in healthy vo…
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FDA approval · 2024-02-06FDA Approved: FABRAZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for FABRAZYME (agalsidase beta) submitted by Genzyme Corporation. FABRAZYME is an enzyme replace…
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CLINICAL_TRIALS trial_result · 2024-01-05Clinical Trial Update: Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease [Phase 1, Phase 2]
Open-label study of AVR-RD-01 for treatment-naive Fabry disease subjects in Phase 1/2 was terminated with results posted.
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CLINICAL_TRIALS trial_result · 2023-12-07Clinical Trial Update: A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 [Phase 1, Phase 2]
A long-term follow-up study of FLT190 in Fabry disease subjects across Phase 1/2 has been completed.
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CLINICAL_TRIALS trial_result · 2023-08-02Clinical Trial Update: Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function [Phase 3]
Phase 3 study comparing PRX-102 to Agalsidase Beta in Fabry disease completed with results posted.
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FDA approval · 2023-06-20FDA Approved: GALAFOLD — Supplemental Application (Amicus Therapeutics US, LLC)
The FDA approved a supplemental application for Galafold (migalastat hydrochloride) from Amicus Therapeutics US, LLC. The action pertains to…
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FDA approval · 2023-05-09FDA Approved: ELFABRIO — Original Application (Chiesi USA, Inc.)
The FDA granted approval for ELFABRIO (pegunigalsidase alfa) from Chiesi USA, Inc. as an original enzyme replacement therapy for Fabry disea…
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CLINICAL_TRIALS withdrawal · 2023-04-07Clinical Trial Update: To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease [Phase 4]
A Phase 4 trial comparing switching from Agalsidase Alfa to Agalsidase Beta in male patients with classic Fabry disease has been withdrawn.
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CLINICAL_TRIALS trial_result · 2023-03-27Clinical Trial Update: Evaluation of the Gastrointestinal Manifestation of Fabry's Disease [NA]
Clinical trial update reporting completed status and posted results for evaluation of gastrointestinal manifestations in Fabry's disease.