Serif’s Modified DNA platform is engineered with carrier molecules and proprietary mRNA cofactors that enhance its activity and expression At the American Society of Gene and Cell Therapy (ASGCT) ...

Best practices most often mentioned by amyloidosis stakeholders included patient-centric multidisciplinary care, responsiveness to patients' questions, and timely intake of new patients who are at ...

Dual pathway blockade targets complement-driven pathology in AChR-positive MG, as well as FcRn-mediated pathogenic IgG recycling. Two patients with refractory anti-acetylcholine receptor (AChR) ...

After the enzyme-linked immunosorbent assay was added to the solid phase red cell adherence test, the detection rate of anti-CD36 increased about 15-fold. Platelet antibodies that may lead to fetal ...

Regenxbio announced plans to pursue accelerated FDA approval of RGX-202 and prepare for a potential commercial launch in 2027. The pivotal portion of the trial of RGX-202, a gene therapy for Duchenne ...

Recurrent atraumatic vertebral fractures warrant serum tryptase, KIT testing, and biopsy at augmentation. Indolent systemic mastocytosis (SM) should be suspected in patients presenting with atraumatic ...

The pathogenic role of CD38-expressing plasma cells in FSGS may be linked to the therapeutic effect, given that circulating CD38+ plasma cell counts have been shown to correlate with disease activity.

At week 52, 50.9% of patients on elafibranor met the primary endpoint of biochemical response compared with 3.8% of those receiving the placebo. Lower baseline alkaline phosphatase (ALP) levels were ...

The FDA granted Fast Track Designation to coramitug for ATTR-CM as it advances in the ongoing phase 3 CLEOPATTRA trial. The US Food and Drug Administration (FDA) has granted Fast Track Designation to ...

Researchers developed an AI-based framework that can generate a report like a primary care physician, which can then help identify undiagnosed patients with ATTR-CM and individuals who are at risk of ...

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