
Innovative therapies for rare diseases such as spinal muscular atrophy (SMA), when paid for by public budgets in the European Union and elsewhere, are often managed by agreements between payers and drug companies, but details can be hard to discern.

Innovative therapies for rare diseases such as spinal muscular atrophy (SMA), when paid for by public budgets in the European Union and elsewhere, are often managed by agreements between payers and drug companies, but details can be hard to discern.

From the decision analysis came an agreement that reimbursement for an orphan drug should be based primarily on its effect on health-related quality of life, its effectiveness, and the availability of other treatment options.

In pediatric patients with frequently relapsing nephrotic syndrome (FRNS), the therapy was well tolerated, but did not lead to significant changes in disease course.

According to researchers, new findings may have important implications for expanding the use of measurable residual disease (MRD) in multiple myeloma.

Investigators identified 5 mutated genes that could serve as biomarkers for disease prognosis and clinical outcomes associated with pancreatic ductal adenocarcinoma, a common cancer with a high mortality rate and poor prognosis.

Rajesh Rajpal, MD, chief medical officer, global head of clinical medical affairs, Johnson & Johnson Vision, discusses the surgical options available to patients undergoing cataract surgery.

Investigators examined medical records spanning 3 decades to look at the effect of poorly differentiated tumor size on outcomes in survival in hepatocellular carcinoma (HCC).

Adherence to continuous positive airway pressure (CPAP) resulted in a large decrease in insomnia and a significant reduction in prevalence of insomnia from baseline to follow-up in patients with obstructive sleep apnea.

Discontinued use and high dosage of alpha-glucosidase inhibitors in combination with metformin was shown to increase risk of psoriasis and psoriatic arthritis in patients with type 2 diabetes compared with those given only metformin.

Crigler-Najjar Syndrome Type 1 is an ultra-rare disease where treatment relies on a liver transplant or full-day phototherapy sessions.

A session at the 5th annual Heart in Diabetes Conference highlights the long-term risks of gestational diabetes.

Reducing the risks of secondary neutropenia, exposure to COVID-19, mortality, and financial toxicity were all major reasons the use of pegfilgrastim biosimilar gained traction during the pandemic, said Kashyap Patel, MD, CEO of Carolina Blood and Cancer Care.

In a recent study, researchers were able to show how different evolutionary forces in aging blood stem cells impact whether or not someone develops acute myeloid leukemia (AML), offering insight into those at higher risk of the disease.

An emphasis on transitions of care should ensure the highest level of care for patients and provide support to physicians.

New research presented at the 5th Heart in Diabetes Conference highlights potential therapeutic options for patients with COVID-19 and diabetes.

Students in grades 3 to 7 who were given eyeglasses as part of a school-based vision program were found to achieve better reading scores and exhibit improved academic achievement over 1 year.

Neuropalliative care is an emerging field for those with conditions such as Parkinson disease, multiple system atrophy, progressive supranuclear palsy, corticobasal syndrome, and other diseases, but access to care remains limited, according to a recent survey.

Data from 2 patient cohorts show that by increasing awareness of such influences as clinic location and patient age and transgender status, gaps in prescription decisions for integrase strand transfer inhibitors (INSTIs) can be lessened.

Results of a large retrospective cohort study showed veterans with chronic headache are at an increased risk of suicide attempts compared with those experiencing other forms of chronic pain.

The Lancet commission aimed to connect stakeholders, raise global awareness of these inequities and disparities, and encourage future research.

David J. Reeves, PharmD, BCOP, of Butler University discusses encouraging results from a recent trial, along with safety questions about the types of patients who weren't studied but are likely to have small cell lung cancer.

After a month of stable utilizations, use of telehealth services dropped 10% in June 2021, according to Robin Gelburd, JD, president of FAIR Health.

Study authors found that knowing a patient’s minimal residual disease status can help pinpoint the right multiple myeloma therapy to improve overall and progression-free survival.

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

In part 2 of a 2-part series, we look at the mental health risks affecting 9/11 rescuers, the lessons learned that apply to health care workers during the current pandemic, as well as continued barriers to care.

The FDA misses the deadline to pull e-cigarette products from the market; Los Angeles public schools require COVID-19 vaccines for those over age 12; rates of still births are on the rise in Mississippi.

Bio-Thera Solutions and Sandoz have partnered for to allow for the commercialization of BAT1706, a bevacizumab biosimilar referencing Avastin, for the treatment of several forms of cancer.

Results of an Australian study highlight the changes that occur in the gut microbiome of pregnant patients with type 1 diabetes.

Patients with BRAF-V600 mutated metastatic melanoma have new treatment options to consider, but scientists are still waiting on additional data to help choose which first-line treatments are best for which patients.

Patients who undergo transplant face an increased risk of skin cancer because of their immunosuppressed status, explained Christine Ko, MD, professor of dermatology and pathology at Yale University.

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