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Implementing artificial intelligence (AI) has transformed clinicians' and health systems' ability to screen for and distinguish forms of cardiorenalmetabolic disease.

Erythropoiesis-stimulating agent (ESA) treatment before or early after regular transfusion therapy improved overall survival (OS) In lower-risk myelodysplastic syndromes (MDS), a new study found.

While the overall effectiveness of collaborative care has been well established, this study aimed to determine which specific components had the most impact.

Phase 2 results reported at the European Lung Cancer Conference show a favorable signal for overall survival for the first-line treatment of KRAS-mutated non–small cell lung cancer (NSCLC). A phase 3 trial is enrolling now.

Medication nonadherence to oral anticoagulants and oral anti–prostate cancer medication has been scrutinized through new research conducted among patients and health care providers and presented by the American Medical Group Association at its 2025 annual meeting, held March 26-29 in Grapevine, Texas.

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients with Duchenne muscular dystrophy (DMD) in the phase 2 FORWARD-53 trial.

For higher-risk multiple myeloma (MM), successful patient selection and monitoring strategies are vital for the management of adverse events and the disease itself.

Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, spoke to the current state of research on revakinagene taroretcel in macular telangiectasia type 2 (MacTel), as well as the importance of patient groups for the rare condition.

Weight loss is a key effect of glucagon-like peptide-1 receptor agonists (GLP-1 RAs), but these drugs may also provide liver health benefits independent of this mechanism.

The European LeukemiaNet (ELN) scoring system showed similar results to the more complicated Integrated Flow Score (iFS) for diagnosing myelodysplastic syndrome (MDS), including in low-risk patients.

A real-world analysis showed a link between continuous glucose monitor (CGM) distribution channel and outcomes for patients with diabetes.

The approval of revakinagene taroretcel (Encelto; Neurotech) addresses a significant unmet need for patients with macular telangiectasia type 2 (MacTel), clinical investigator Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, said.

Ajai Chari, MD, stresses the importance of making bispecific therapies more accessible at the community level to improve outcomes in multiple myeloma.

Resmetirom offers a targeted approach to metabolic dysfunction–associated steatohepatitis (MASH) for use alongside lifestyle modifications.

Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, discusses data supporting the FDA approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals), the first and only therapy indicated for the treatment of macular telangiectasia type 2 (MacTel).

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.

Inclusion 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.

Lipoprotein(a) [Lp(a)] plays a key role in assessing cardiovascular risk, making awareness efforts equally essential for prevention and early intervention.

In 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.

Ajai Chari, MD, University of California San Francisco, explores the challenges that accompany integrating chimeric antigen receptor (CAR) T-cell therapies into multiple myeloma treatment.

Patients who did not complete a distress screener were also less likely to receive autologous stem cell transplants.

The PROMPT trials evaluate the influence of electronic health alerts for informing appropriate, evidence-based therapies to patients with cardiovascular conditions.

The rate of cGVHD-free survival was 78% at 1 year in patients who received Orca-T compared with 38% among patients who received standard allogeneic stem cell transplant for hematologic malignancies.

Tenacious 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.

The 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.