Specialty Pharmacy

Results from the phase 3 SAPPHIRE trial show significant improvements in motor function in children and adults with spinal muscular atrophy.

If long-term efficacy results are positive, metformin can become an accessible, affordable treatment option for autosomal dominant polycystic kidney disease (ADPKD).

A systematic review and meta-analysis revealed key insights into lipid parameters in patients with systemic sclerosis.

State policymakers should target bad actors while protecting access.

High-density lipoprotein cholesterol, known for protective cardiovascular effects, could play a role in reducing the risk of stress urinary incontinence.

The complete response letter (CRL) stated that vatiquinone's new drug application (NDA) did not provide substantial evidence of efficacy and would need to be evaluated in a new trial.

Unicycive Therapeutics secures a US patent for UNI-494, a promising treatment for chronic kidney disease, enhancing its development and partnership potential.

By improving low-density lipoprotein cholesterol (LDL-C) levels, statins reduced complications after pipeline embolization device implantation for intracranial aneurysms.

New research reveals that novel biomarkers enhance risk prediction for kidney failure and mortality in chronic kidney disease (CKD), paving the way for personalized treatment.

Multiple FDA-approved drugs without lipid-lowering indications were identified to have potential lipid-lowering properties.

The researchers speculate that this is because of the antioxidant and anti-inflammatory properties of coffee and tea.

Designed for use in Celiac disease, larazotide was found to hasten resolution of multisystem inflammatory syndrome symptoms in children with the condition.

Brad Cochran of Cardinal Health explains the One Voice Initiative, which aims to bolster independent pharmacies.

Mona El-Mouwfi explores how pharmacists enhance hydroxyurea therapy for sickle cell disease, improving patient outcomes and addressing treatment barriers effectively.

Intravenous immunoglobulin (IVIG) is a vital treatment for immunodeficiency and autoimmune disorders, with diverse mechanisms, brand-specific differences, and patient-centered considerations shaping safe and effective use.

The approval builds upon concizumab’s previous December 2024 clearance and allows for treatment in patients with hemophilia A or B, both with or without inhibitors.

Ivermectin shows promise in reducing malaria cases, enhancing vector control, and highlighting pharmacists' vital roles in public health strategies.

Namrata Shah, MD, highlights lenacapavir’s ease of use, long-acting protection, and the vital role pharmacists play in expanding equitable HIV prevention access.

The treatment proved efficacy across all 3 key markers of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

Tildrakizumab 100 mg shows significant efficacy in treating psoriatic arthritis, achieving key clinical trial end points and offering hope for patients.

Hospitals and health systems are adapting specialty and infusion pharmacy strategies to meet evolving payer mandates, rising costs, and shifting care models by expanding biosimilar use, optimizing prior authorizations, and integrating technology to enhance patient access and operational efficiency.

A phase 2 trial shows oxylanthanum carbonate (OLC) effectively manages hyperphosphatemia in patients with chronic kidney disease (CKD), offering a promising treatment option.

Sepiapterin treatment allowed patients to liberalize their diet while reaching or exceeding recommended daily protein allowances in patients living with phenylketonuria.

Independent specialty pharmacies face growing financial and operational pressures from rising drug costs, restrictive PBM contracts, and regulatory complexity, prompting many to explore strategic partnerships or sales as a means to sustain clinical excellence, preserve autonomy, and ensure long-term viability in an increasingly challenging health care environment.

Namrata Shah, MD, discusses the groundbreaking efficacy and patient-centered potential of twice-yearly lenacapavir for HIV preexposure prophylaxis (PrEP), supported by pivotal data from the PURPOSE trials.

The decentralization of US health care, accelerated by virtual care adoption, shifting reimbursement models, and a growing demand for complex therapies, is elevating the role of specialty and infusion pharmacies as essential, tech-enabled partners in delivering coordinated, patient-centered care across outpatient and home-based settings.

High levels of remnant cholesterol were associated with increased risk of developing metabolic dysfunction-associated steatotic liver disease (MASLD) independent of low-density lipoprotein cholesterol (LDL-C).

The approval represents a major advancement for patients with moderate-to-severe chronic hand eczema, as delgocitinib becomes the first treatment available for this population in the US.