Clinical

At the time of treatment, there was just 1 paper documenting the use of nusinersen in a case of more mild type 0 spinal muscular atrophy(SMA).

Chimeric antigen receptor (CAR) T cells can be highly effective, but the durability of the therapy has been lacking in many patients with hematological malignancies. Many efforts are underway to fix this problem.

The FIDELIO-DKD program is perhaps just the beginning of seeing how finerenone can be used, said Rajiv Agarwal, MD, MS, FASN, a professor of medicine at Indiana University School of Medicine and a staff physician at the Veterans Affairs Medical Center in Indianapolis, Indiana.

Researchers have identified additional mutations in both SMN1 and other genes, some of which may be associated with the onset of spinal muscular atrophy (SMA).

New research suggests it’s not disruptive to give radiation therapy to patients who are awaiting CAR T-cell therapy for relapsed or refractory multiple myeloma.

Patricia K. Coyle, MD, of Stony Brook University Neurosciences Institute, explains how studies are trying to answer a debate about 2 approaches to treat multiple sclerosis (MS).

Treatment targets include several interleukins, immunoglobulin E, and alarmins, pointed out Nicola Hanania, MD, MS, pulmonary critical care physician and director, Airway Clinical Research Center, Baylor College of Medicine, Houston.

In patients under age 70, lung cancer conferred the highest risk of death from coronavirus disease 2019 (COVID-19).

Black individuals with diabetic macular edema (DME) exhibited a significantly lower likelihood of visual acuity improvement following intravitreal bevacizumab treatment compared with White and Hispanic patients.

The liquid serum assay was cleared for use as a companion diagnostic for olaparib to identify patients with BRCA1/2- and/or ATM-mutated metastatic castration-resistant prostate cancer.

FDA approves a coronavirus disease 2019 (COVID-19) antibody treatment; North Dakota braces hospitals for surging cases; fruit consumption among children is low.

Pfizer said interim data on its coronavirus disease 2019 (COVID-19) vaccine show 90% efficacy; meanwhile, President-elect Joe Biden released the names of his coronavirus advisory board.

Patients with chronic migraine who received a combination therapy of erenumab and onabotulinumtoxinA exhibited significant reductions in monthly headache days and monthly migraine days.

Understanding these genetic variants discovered through sequencing efforts is key to further progress in clinical genetics.

Lametra Scott, PharmD, CCHP, CEO of Breaking The Sickle Cell Cycle Foundation Inc discusses the current standard of care in sickle cell disease (SCD) and the unmet needs facing patients.

Categorizing by eosinophilic/noneosinophilic subtype enables better targeted treatment, noted Anne Reihman, MD, third-year pulmonary and critical care fellow, University of Colorado, Division of Pulmonary Sciences and Critical Care Medicine.

There did not appear to be any particular patient characteristics linked with best responses to carfilzomib in patients with multiple myeloma, with the exception of low serum lactate dehydrogenase.

Immune-associated neurotoxicity in patients following chimeric antigen receptor (CAR) T-cell therapy may be due to monocyte-like cells in infusion products, explained Michael R. Green, PhD, University of Texas MD Anderson Cancer Center.

Presenters at CHEST 2020 discussed the concept of how patients with interstitial lung disease (ILD) can develop a progressive fibrosis phenotype and how providers can detect and treat these patients.

Two CHEST 2020 abstracts looked at mortality risks for patients with chronic obstructive pulmonary disease (COPD).

Jill Jarecki, PhD, chief scientific officer at Cure SMA explained the current data and potential for gender to impact the severity spinal muscular atrophy (SMA).

The procedure might work best in patients who have had multiple sclerosis (MS) for less than 10 years, the report said.

The study compared patients given-low-dose ketamine with those given morphine for acute pain relief.

Researchers conducting broad next-generation sequencing discovered more actionable variants in patients and families than they would have otherwise; they did so by using a universal approach, rather than sticking with clinical guidelines.

Quality of life for patients with multiple sclerosis (MS) could be better when they are off medication because they will not have adverse effects related to those medications, said John Corboy, MD, professor of neurology, University of Colorado Denver, School of Medicine.