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This new analysis of 5-year data on a novel gene therapy from PTC Therapeutics shows both cognitive and motor function improvement in children with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare central nervous system disorder.

Bevacizumab biosimilar LY01008 demonstrated comparative safety, efficacy, immunogenicity, and pharmacokinetic profiles to the reference product Avastin when combined with paclitaxel and carboplatin in patients with non–small cell lung cancer (NSCLC).

A review of instruments used to assess upper-limb function in patients with neuromuscular diseases found that more tools are needed to address specific diseases.

Secukinumab was associated with a high rate of short- and long-term drug survival in the treatment of patients with psoriasis, with factors such as obesity and prior biologic use linked to discontinuation of use.

Artificial intelligence (AI) was used to predict response of high-grade extrauterine serous carcinoma (HGSC) to adjuvant platinum chemotherapy.

New research presented at IDWeek 2021 presented data on the microbiota-based live biotherapeutic RBX2660 for recurrent Clostridioides difficile infection.

Results of a meta-analysis show migraineurs suffer from poorer sleep quality than healthy controls.

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder; a new report said that plasma lyso-sphingomyelin levels can be used to not only diagnose the rare disease but also predict severity and type.

Coverage of our peer-reviewed research and news reporting in the health care and mainstream press.

In an interview for ESC Congress 2021, Deepak L. Bhatt, MD, MPH, executive director of interventional cardiovascular programs at Brigham and Women’s Hospital Heart & Vascular Center and professor of medicine at Harvard Medical School, describes the treatment outlook for patients with heart failure with preserved ejection fraction (HFpEF).

Investigators reported comprehensive genomic features of patients with clear cell renal cell carcinoma, potentially giving providers a better understanding of the molecular features associated with the disease.

Dosing from pivotal phase 3 trials of tyrosine kinase inhibitors (TKIs) in chronic myeloid leukemia (CML) is not optimal for real-world patients who experience different efficacy and adverse events (AEs).

Maralixibat is the first treatment approved for cholestatic pruritus in patients with Alagille syndrome who are 1 year or older.

A new study suggests the lower-cost, more patient-friendly assessment method may be a useful tool for clinicians.

A large study out of the United Kingdom finds that women who use hormone replacement therapy (HRT) do not have a higher risk of dementia.

Highlighting the latest ophthalmology-related news reported across MJH Life Sciences™.

Current knowledge about how Parkinson disease (PD) affects patients from different socioeconomic or ethnic groups is lacking, said The Michael J. Fox Foundation for Parkinson’s Research (MJFF), and diverse groups are underrepresented in research.

A recent review explored issues in diagnosing and treating children with growth hormone deficiency (GHD).

Although physicians’ clinical decisions serve as the biggest drivers behind the cost of care, hospitals have long been reluctant to take financial accountability. If such accountability is to be transformed from a diffuse fear to a manageable managerial task, institutional engagement with physicians will be a critical next step.

Chronic disease caregivers are more likely to report both risky and health-promoting behaviors than noncaregivers, according to a new study.

The CDC strongly recommends pregnant people get vaccinated against COVID-19; Eli Lilly pledges to reduce the price of its insulin lispro injection; House Democrats are divided over Biden’s health care spending bill.

Results of a 15-year cohort study found waist to height ratio was associated with increased risks of severe diabetic eye disease among individuals with type 1 diabetes.

Patients with multiple sclerosis (MS) who had a higher education level experienced less cognitive impairment related to their disease than patients with a lower education level, investigators concluded.

This analysis of Veterans Aging Cohort Study participants highlighted 2 factors that may predispose persons living with HIV to a higher risk of sudden cardiac death.

Deepak L. Bhatt, MD, MPH, outlines the benefits of starting patients on sodium glucose co-transporter 2 (SGLT2) inhibitors while they are hospitalized.

These second-generation inhibitors include acalabrutinib, zanubrutinib, and tirabrutinib, and researchers hope they can overcome the off-target toxicity and treatment resistance that can be experienced with ibrutinib.

Patients with a positive fecal immunochemistry test (FIT) were shown to be at significantly greater risk of developing psoriasis than those with FIT-negative tests.

Findings from this new study are the result of an investigation into over 360 children, many with active hepatitis, elevated alanine aminotransferase levels, and an immune active phenotype.