Building Community and Advancing Care at MDA 2025
One of the major highlights of the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is that invaluable networking opportunities can help clinicians elevate their own best practices.
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MASH Clinical Trials Historically Underrepresent Certain Groups
March 24th 2025Inclusion of female and Hispanic/Latino patients has increased over time, but most trials of metabolic dysfunction-associated steatohepatitis (MASH) still take place in high-income countries and have majority-White patient populations.
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FDA Approves Vutrisiran to Mitigate Cardiomyopathy-Related Risks and Conditions
March 21st 2025In a recent decision, the FDA approved vutrisiran (amvuttra), making it the only approved therapy for adults with hereditary transthyretin-mediated amyloidosis (ATTR-CM) or wild-type cardiomyopathy.
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Streamlining "Brain-to-Vein" Time, Patient Selection, Imperative for Implementing CAR T for MM
March 21st 2025Ajai Chari, MD, University of California San Francisco, explores the challenges that accompany integrating chimeric antigen receptor (CAR) T-cell therapies into multiple myeloma treatment.
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Neurologists Share Tips for Securing Patient Access to Gene Therapies
March 19th 2025Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular diseases.
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Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
March 18th 2025The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but also logistical hurdles and a need for complex discussions between clinicians and families.
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Solving the Transition Conundrum as More Children With Muscular Dystrophy Live to Adulthood
March 17th 2025Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach adulthood thanks to transformative therapy advances.
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As Claims Denials Surge, AI and Data-Driven Insights Equip Clinicians, Hospitals to Fight Back
March 13th 2025As health care costs continue to rise and the burden of chronic disease grows, data-driven insights will be essential in shaping the future of patient care, according to experts from Komodo Health and SmarterDx.
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Demographic Disparities in Video Visit Telemetry: Understanding Telemedicine Utilization
March 7th 2025A stratified demographics analysis of video visit telemetry data reveals that age older than 65 years and African American/Black race are associated with higher video visit failure rates, whereas language, sex, and ethnicity are not.
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More than 5 years after the American Society of Clinical Oncology warned of emerging disparities in precision medicine, efforts by community practices to embrace technology, form partnerships, and use data show how patients can gain access to personalized approaches. But challenges remain, especially for those covered by Medicaid.
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First-Line Tislelizumab Plus Chemotherapy Approved in ESCC Expressing PD-L1
Published: March 4th 2025 | Updated: March 4th 2025Tislelizumab plus chemotherapy received FDA approval as a frontline therapy for individuals with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1.
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Survey Explores Needs of Patients With DMD, BMD Transitioning to Adulthood
February 28th 2025Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD), but those who had more siblings reported being more ready to transition to adult life.
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Potential Spillover Effects on Traditional Medicare When Physicians Bear Medicare Advantage Risk
Medicare beneficiaries treated by physicians with high levels of Medicare Advantage risk exposure had higher care quality and efficiency outcomes compared with those treated by other physicians.
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NGS-Based Test Accurately Detects Post–Allo-HSCT Relapse in AML, MDS
February 21st 2025The next-generation sequencing (NGS)–based AlloHeme test accurately predicted relapse following allogeneic hematopoietic stem cell transplantation (allo-HSCT) in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
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