Identifying Risk to Enable Early Detection of Type 1 Diabetes
March 19th 2024An expert reviews risk factors for type 1 diabetes including autoimmune disease and family history, along with the role of autoimmune antibody screening to enable early disease detection, though access to these specialized tests can be limited.
Strategic Insights Into Evolving Gene Therapy Trends
March 19th 2024Emma Ciafaloni, MD, FAAN, and Jessica Nance, MD, MS, discuss promising developments in gene therapy, highlighting long-term efficacy, enhanced target effects, and the potential impact of early screening on treatment outcomes for genetic diseases.
Prior Authorization and Access to Targeted Treatment for Ph+ ALL Patients
March 18th 2024Jonathan Abbas, MD, compares the safety and effectiveness of different targeted agents and considerations when selecting a frontline regimen for adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).
Duchenne Muscular Dystrophy Treatment Landscape Review
March 12th 2024Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early intervention and comprehensive care.
Driving Clinical Insights: Harnessing Functional Assessments in DMD Research
March 12th 2024Jessica Nance, MD, MS, discusses the significance of surrogate end points, like functional assessments, in clinical trials of treatments for Duchenne muscular dystrophy and their implications for clinical benefits.
Diagnosis and Treatment Timeline: Different Phases of Ph+ ALL Management
March 11th 2024The panel provides an overview of the various Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) treatment phases, highlighting the important discussion around inpatient versus outpatient care.
Future of Myelofibrosis: Anticipated Data and Emerging Therapies
March 6th 2024Aaron Gerds, MD, MS, explores the future landscape of myelofibrosis with insights from the 2003 ASH Annual Meeting and Exposition (ASH 2023), and delves into anticipated data on combination therapies and ongoing trials shaping the next phase of myelofibrosis treatment.
Review of Clinical Evidence for Delandistrogene Moxeparvovec for DMD
March 5th 2024Jessica Nance, MD, MS, reviews the data and evidence that led to accelerated approval of delandistrogene moxeparvovec-rokl for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.