Diagnosis & Disease Information

gene therapy research

Enzyme Replacement Therapy May Stabilize Pompe Disease Gene Expression

Transcriptomic gene analysis of skeletal muscle biopsies from patients with late-onset Pompe disease (LOPD) suggests that enzyme replacement therapy attenuates the expression of dysregulated genes involved in lysosomal function, according to a recently published study in Molecular Genetics and Metabolism. Although enzyme replacement therapy has significantly improved the survival and quality of life of many patients…

fatigued woman

Sutimlimab Improves Fatigue and Quality of Life Among Patients With CAD

Researchers reported that sutimlimab provides sustained improvements in patient-reported outcomes and quality of life in patients who have been diagnosed with cold agglutinin disease (CAD), according to a study published in Blood. CAD is a rare autoimmune hemolytic anemia mediated by the classical complement pathway. The most commonly reported symptoms are fatigue and decreased quality…

hemoglobin analysis

New Study Supports the Efficacy of Sutimlimab in CAD Treatment

The use of sutimlimab in the treatment of patients with cold agglutinin disease (CAD) resulted in continued rapid and sustained improvements in hemoglobin (Hb) level, hemolytic markers, and quality of life during Part B of the phase 3 Cadenza study, according to a poster abstract published in Blood. The randomized, double-blind, placebo-controlled study reported on…

pompe disease experimental therapies

Select Patients With PD Do Not Respond to Alglucosidase Alfa

Avalglucosidase alfa has a positive clinical effect in patients with infantile-onset Pompe disease (IOPD) whose condition worsened while being treated with alglucosidase alfa. This is according to preliminary results from a phase 2 clinical trial published in Genetics in Medicine.  The aim of the open-label, ascending dose trial was to evaluate the safety, efficacy, and…

covid-19 vaccine

Response to COVID-19 Vaccine Not Impaired in Patients With CAD on Sutimlimab

Data from the phase 3 Cardinal and Cadenza studies have shown that the response to coronavirus disease 2019 (COVID-19) vaccinations was not impaired in patients with cold agglutinin disease (CAD) who were undergoing treatment with sutimlimab. The study, published in Blood, also noted that sutimlimab treatment was effective and well tolerated among the patients. “In…

fatigued woman

Patients With CAD Report Improved Outcomes After Sutimlimab Treatment

An analysis of patient-reported data from the CARDINAL Part A trial has revealed substantial improvements in fatigue symptoms and quality of life (QOL) in patients with cold agglutinin disease (CAD) receiving sutimlimab (Enjaymo™). “Sutimlimab rapidly inhibits classical [complement pathway (CP)] activity and subsequent hemolysis, which coincides with rapid, sustained improvements in fatigue symptoms and overall…

FDA-Priority-Review

FDA Grants Priority Review to Efanesoctocog Alfa for the Treatment of Hemophilia A

Sanofi Global Health announced that the US Food and Drug Administration (FDA) has granted priority review to the Biologics License Application (BLA) for efanesoctocog alfa, a novel investigational recombinant factor VIII replacement therapy for people with hemophilia A. The review, announced by news release, has an FDA target decision date of February 28, 2023. “Factor…

european commission

European Commission Approves Avalglucosidase Alfa for Pompe Disease

The European Commission approved avalglucosidase alfa, sold under the brand name Nexviadyme® by Sanofi, as a potential new standard of care for infantile-onset and late-onset Pompe disease. This makes avalglucosidase alfa a new treatment option for patients in Europe who have had Pompe disease for more than 15 years. “The approval of Nexviadyme in Europe…

clinical trial research

Studies Testing Potential Treatment for MS and MG Put on Partial Clinical Hold

Phase 3 clinical trials testing the drug candidate tolebrutinib in multiple sclerosis (MS) and myasthenia gravis (MG) have been put on partial clinical hold by the US Food and Drug Administration (FDA) due to treatment-induced liver injury in some patients. As a result, new patient recruitment into the trials has been paused in the United…

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