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Both cycling and home-based exercise training helped maintain gait and balance parameters in children with Duchenne muscular dystrophy (DMD), with cycling training also improving antero-posterior balance.

A retrospective, single-center cohort study utilized liver ultrasound, serum collection, and proteome analysis to draw associations between spinal motor neuron (SMN) protein depletion and heightened risk for fatty liver disease in spinal muscular atrophy (SMA).

The 10th annual meeting of the Family Heart Global Summit will bring together leading scientists and practitioners with patients to discuss treatment of inherited lipid disorders.

The majority of federal savings indicated by the Inflation Reduction Act's Drug Price Negotiation Program can be attributed to 3 drugs.

Changes in formulary coverage for 2024 are impacting Medicare patients' access to and ability to afford various prescription medications.

Though circulating high mobility group box-1 levels appear to be a biomarker for pulmonary arterial hypertension (PAH) in adults with congenital heart disease, the same is not true in pediatric patients.

A meta-analysis demonstrated the superiority of Bruton tyrosine kinase inhibitors (BKTi) in the overall survival of patients with chronic lymphocytic leukemia compared with other therapies.

A review of the upcoming schedule of the Institute for Value-Based Medicine.

Analysis of the phase 3 explorer7 (NCT04083781) study indicates a preference for concizumab prophylaxis over no prophylaxis in patients with hemophilia A or B with inhibitors.

Jennifer Cocohoba, PharmD, MAS, AAHIVP, University of California, San Francisco, School of Pharmacy, discusses the benefits of pharmacy-administered long-acting injectable antiretroviral therapy for HIV and patient attitudes toward pharmacy-administered treatment.

Recent years have brought a more targeted approach to treating acute myeloid leukemia (AML) based on accumulating knowledge about the genetic makeup of the heterogenous disease.

CAR T-cell therapy results in a high incidence of cytokine release syndrome, but it is manageable in most cases.

A predictive tool considering disease duration, age at disease onset, age, and Expanded Disability Status Scale score estimated patients’ risk of transition to secondary progressive multiple sclerosis (MS).

Lumbar spine bone mineral density improved significantly and was maintained during long-term tumor necrosis factor inhibitor (TNFi) treatment for axial spondyloarthritis.

A codesigned online nutrition program could potentially fill a need for reliable and evidence-based nutrition information for patients with multiple sclerosis (MS).

Ravulizumab therapy may enable patients with paroxysmal nocturnal hemoglobinuria (PNH) to comparable achieve activity levels and patient-reported outcomes to the US general population.

This article first appeared on The Center for Biosimilars®.

This investigation was conducted among older, frail patients, a population typically underrepresented in clinical trials.

The FDA has approved axatilimab-csfr (Niktimvo) for treating adults and children with chronic graft-versus-host disease who have progressed on at least 2 prior treatments.

Caitlin Kindberg, a patient advocate from Nashville, Tennessee, discusses her experience finding proper treatment for idiopathic hypersomnia and the challenges associated with living with this condition.

Study results demonstrated significant correlations between fatigue and reductions in walking speed and mobility among patients with multiple sclerosis.