The American journal of managed care
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Just 3 disease-modifying treatments-edaravone, riluzole, and sodium phenylbutyrate and taurursodiol (PB/TURSO)-are currently FDA approved to slow progression of amyotrophic lateral sclerosis (ALS). A fourth therapy has been recently approved under accelerated approval and is contingent upon verification of clinical benefit in confirmatory trials(s). Therapy selection is based largely upon patient characteristics, as guidelines have not been updated since the recent approval of PB/TURSO or accelerated approval of tofersen. ⋯ Although evidence is lacking for many pharmacologic therapies, providers use symptomatic treatments to address common symptoms including anxiety, depression, emotional lability (pseudobulbar affect), fasciculations, fatigue, insomnia, muscle cramps or spasms, musculoskeletal pain due to immobility, neuropathic type pain, excessive salivation (sialorrhea), spasticity, constipation, and urinary urgency. Emerging agents offer some hope for patients with ALS. Among the drugs, biologics, and interventions under investigation for ALS are an oral tyrosine kinase inhibitor, RIPK1 inhibition, the use of mesenchymal stem cells, antisense oligonucleotides, sequential administration of all experimental treatments in a new study design, and modification of the patient's own mesenchymal stem cells.
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Neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME) can cause substantial disease burden for patients. Several organizations have published clinical guidelines on appropriate diagnosis and treatment recommendations to alleviate this burden. Treatment approaches include both nonpharmacologic methods and pharmacologic therapies, with anti-vascular endothelial growth factor (VEGF) therapy being the standard of care. ⋯ Emerging treatments and dosing strategies aim to decrease treatment burden and increase patient safety. Retina specialists can play a key role in improving the management of both nAMD and DME by incorporating patient-specific treatment strategies tailored to improve clinical outcomes. Enhanced knowledge of retinal disease therapies will allow clinicians to optimize evidence-based treatment strategies to improve clinical outcomes for their patients.
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Amyotrophic lateral sclerosis (ALS) is a fatally progressive degenerative disease, with many patients succumbing to the condition within 3 to 5 years after diagnosis. It is a rare, orphan disease with an estimated US prevalence of 25,000 patients. Patients with ALS and their caregivers are faced with a substantial financial burden as a result of the condition, as ALS has an estimated national financial burden of $1.03 billion. ⋯ Other recently approved therapies include oral edaravone, a combination therapy of sodium phenylbutyrate and taurursodiol (PB/TURSO), and tofersen, which is administered intrathecally and approved under an accelerated approval. Long-term studies have shown PB/TURSO to have a dual benefit on survival and function. The Institute for Clinical and Economic Review (ICER) 2022 Evidence Report for ALS does not value the high price points of edaravone and PB/TURSO as cost-effective based on the current evidence, despite valuing the need for new treatment options for this patient population.
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To compare the frequency of self-reported gaps in care coordination and self-reported preventable adverse events among adults with vs without diabetes. ⋯ Interventions to improve quality of care for patients with diabetes could incorporate patient-reported gaps in care coordination to aid in preventing adverse events.