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Music Therapy May Benefit Adults With Sickle Cell Disease

Patients with sickle cell disease (SCD) may experience beneficial effects from music therapy (MT), according to the results of a new study published in the Journal of Pain Research. Patients who received MT learned new self-management skills and also improved their ability to cope with pain. The study found that MT significantly improved scores in…

Augmentation Therapy Prescribed in AATD Despite Limited Clinical Evidence

Researchers reported that a significant number of patients with alpha-1 antitrypsin deficiency (AATD) with genotypes Pi*SZ and Pi*MZ were prescribed augmentation therapy (AT) despite a lack of definitive evidence of efficacy in these genotypes, as published in the International Journal of Chronic Obstructive Pulmonary Disease. AT is the only licensed treatment available for a lung…

MTC Survivors May Be Disproportionately Affected by COVID-19

A web-based survey concluded that thyroid cancer survivors experienced increased anxiety during the COVID-19 pandemic when compared to the prepandemic era, as published in Endocrine Practice. Most (83%) respondents indicated that their lives were very different during the pandemic. The study enrolled 378 responders who met all inclusion criteria. These included patients diagnosed with thyroid…
elementary school students

SCD Independently Associated With Impaired Cognitive Function

Sickle cell disease (SCD) is known to be associated with a higher prevalence of sensorineural hearing loss (SNHL) and poor school performance compared to hemoglobin AA carriers, increasing the risk for cognitive impairment in these patients. Nonetheless, hearing impairment in the absence of SCD is also negatively associated with learning-related cognitive functions, leaving the association…
muscle cell tissue

Muscle Shear-Wave Elastography Useful for Monitoring DMD Progression

Ibrahim and Ayşe explored the application of muscle shear-wave elastography (SWE) as an adjunct clinical tool in monitoring pediatric musculoskeletal disorders such as Duchenne muscular dystrophy (DMD). Their study is part of a book published under the working title of Elastography – Applications in Clinical Medicine. Magnetic resonance imaging elastography and ultrasound elastography are technological…

Characteristics Found in Novel AATD Mutation Could Help Advance Treatment

Researchers in Germany described a new genetic variant causing alpha-1 antitrypsin deficiency (AATD) called PiQ0Heidelberg, as published in Respiratory Medicine Case Reports. This variant was previously identified but not clinically described. The patient was a 77-year-old woman who was classified with Global Initiative for Chronic Obstructive Lung Disease grade 3. She also had group B…

Researchers Highlight Pediatric NMOSD Cases to Help Improve Prognosis

Most pediatric cases of neuromyelitis optica spectrum disorder (NMOSD) differed from adult cases due to antibody destruction of myelin oligodendrocyte glycoproteins (MOGs) as opposed to anti-aquaporin-4 (AQP4) autoantibodies, according to results from a study published in Multiple Sclerosis and Related Disorders. Both AQP4 and MOG antibodies necessitated the constant implementation of immunosuppressive treatments to improve…

Different Prostacyclin Analogs Can Help Treat Pediatric PAH

Researchers provided a comprehensive overview of the advantages and drawbacks of prostacyclin and its analogs for the treatment of pulmonary arterial hypertension (PAH) in pediatric patients, as published in European Review for Medical and Pharmacological Sciences. “Epoprostenol can be effective against severe pulmonary hypertension,” the authors said. Iloprost, on the other hand, can treat severe…

Mortalin Inhibition May Have Therapeutic Potential in MTC

Analogs of the heath shock protein 70 (HSP70) inhibitor MKT-077 can suppress human medullary thyroid carcinoma (MTC) cells in culture and mouse xenografts, according to results of a new study published in the International Journal of Molecular Sciences. MKT-077 is a water-soluble rhodocyanine dye analog. Previous studies have shown that it can inhibit mortalin, a…

Numeric Rating Scale Could Assess NMOSD Spasticity

Self-reported scores on the Numeric Rating Scale (NRS) from patients with neuromyelitis optica spectrum disorder (NMOSD) and multiple sclerosis (MS) were correlated with physicians’ scores on the Modified Ashworth Scale (MAS) (r =.934; P <.001), as published in Multiple Sclerosis and Related Disorders. “Patients’ self-reported NRS score had a good correlation with clinician-evaluated MAS score…
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