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A child with profound genetic hearing loss was dosed with the investigational otoferlin gene therapy (DB-OTO), becoming the first patient dosed in a phase 1/2 trial evaluating the treatment, and experienced improved auditory response at week 6 with no concerning safety signals.
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A retrospective study presented at the 2023 American Association of Neuromuscular and Electrodiagnotsic Medicine meeting revealed that social determinants of health (SDOH) are linked to poorer treatment outcomes in patients who have myasthenia gravis, emphasizing the need for identifying and supporting at-risk individuals.

For patients with chronic kidney disease, dose adjustments for other medications requires coordination with other specialists to maintain treatment efficacy, explained Linda Awdishu, PharmD, FASN, professor and division head of clinical pharmacy at the University of California, San Diego, Skaggs School of Pharmacy and Pharmaceutical Sciences.

Smaller noncoding RNA is being used in clinical practice and as knowledge grows of long noncoding RNA, use of it should follow, explained Laura Denby, PhD, Kidney Research UK Senior Fellow, senior lecturer and principal investigator in the Centre for Cardiovascular Science at the University of Edinburgh.

Because of their study's findings, the researchers urged clinicians to screen patients with atopic dermatitis (AD) for cognitive dysfunction.

Two posters presented at Kidney Week 2023 evaluated the global state of chronic kidney disease (CKD) and shed light on delays in CKD diagnoses in the United States.

A study evaluating disparities among children with acute leukemia found that Black children are more likely to present with higher disease burden at diagnosis compared with non-Hispanic White children.

Researchers highlighted the potential long-term consequences of COVID-19 on incident autoimmune and autoinflammatory connective tissue disorders in a recent study.

Sickle cell disease (SCD) has known complications that include organ damage and failure, rhabdomyolysis, glaucoma, splenic sequestration, and vaso-occlusive crises. At present, the only potential cure is a bone marrow transplant, but it can be difficult to find a donor and there is a high rejection risk.

While response and safety may vary across racial and ethnic subgroups, progression-free survival and overall survival does not appear to differ when chimeric antigen receptor T-cell therapy is used in the treatment of patients with multiple myeloma.

Despite e-cigarette use dropping 4.1% in the past year, it is still the most commonly used tobacco product among both middle and high school students.

Amy Laughlin, MD, MSHP, of Orlando Health gave a preview of the topic she is presenting on at today's American Journal of Managed Care®’s Institute for Value-Based Medicine® event in New York City.

The new vaccine program will protect against influenza and COVID-19.

High glucose variability in children and young adults with type 1 diabetes (T1D) strongly predicted slowed nerve conduction velocity, a forerunner of diabetic peripheral neuropathy, in a recent study.

The drug’s mode of action is different than other types of corticosteroids because it’s based on the differential effects on glucocorticoid and mineralocorticoid receptors.

After implementing a multidisciplinary surgical approach, researchers found that use of the new approach, residual disease, and age were all independent predictors of overall and progression-free survival for patients with ovarian cancer.

Immune thrombotic thrombocytopenic purpura (TTP) can be treated safely and effectively with caplacizumab.

Key opinion leaders hold a discussion highlighting significant factors in heart failure staging.

A panel of medical experts discuss various categories of heart failure and their clinical implications.

A panel of experts agreed that exa-cel, a sickle cell disease gene therapy, was safe enough for clinical use, setting the stage for a potential FDA approval; the United States saw an increase in infant mortality rates for the first time in more than 2 decades; the new antiburnout campaign from the CDC asks leaders to better support health care workers.

Patients with colorectal cancer (CRC) can use C-reactive protein (CRP), lymphocyte to CRP ratio, and CRP to albumin ratio as markers for CRC prognosis.

The addition of durvalumab to first-line chemotherapy, followed by maintenance treatment with durvalumab plus olaparib significantly improved progression-free survival in patients with newly diagnosed advanced or recurrent endometrial cancer, according to data from the phase 3 DUO-E/GOG-3041/ENGOT-EN10 trial.

Central nervous system (CNS) progression was reduced with osimertinib plus chemotherapy for patients with EGFR-mutated non-small cell lung cancer plus CNS metastases.

Investigators evaluated the utility of the MG Symptoms Patient-Reported Outcome (PRO) within the armamentarium of instruments that currently evaluate myasthenia gravis (MG) severity.

No genes were identified in psoriatic patches that were not also mutated in the patient’s unaffected skin area.

The phase 3 FLAMES trial results demonstrated an improvement in progression-free survival with senaparib monotherapy vs placebo, regardless of patient subgroup, in patients with newly diagnosed, advanced ovarian cancer.














