Jared Kaltwasser

The company said it was optimistic that the issues identified by the Food and Drug Administration could be resolved with new data.

Healthcare organizations are finding that their data foundations are not as solid as they expected.

In an opinion article, investigators argue that gene therapies may not achieve their full potential if the innate inflammation in Duchenne muscular dystrophy is not effectively managed.

Absent a federal committee’s recommendation, Duchenne muscular dystrophy advocates are trying to get states to adopt screening programs.

Many parents said they were afraid of noninvasive ventilation, with some citing inaccurate concerns about ventilation devices.

Investigators found that making a few changes to a common patient-reported outcomes assessment improved its validity in patients with Duchenne muscular dystrophy.

Medicare Advantage plans have drawn the ire of lawmakers for aggressive coding. Investigations are underway. Some hope artificial intelligence (AI) can be part of the remedy.

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study suggests.

Muscle weakness has the biggest impact on patients’ ability to walk, but other factors matter too, researchers found.

Capricor officials said the company was prepared for the meeting and does not expect it to delay the FDA’s decision on the therapy.

Nirvana, a healthcare technology startup, uses artificial intelligence to better predict insurance coverage.

A new report suggests combining the antimalarial drug with aminoglycosides could enhance the latter’s efficacy, which could also have important safety implications.

A trial found magnetic resonance imaging correlated with physical function tests in patients with Duchenne muscular dystrophy.

A new meta-analysis finds corticosteroids have long-term benefits, and a separate study suggests benefits hold regardless of dosing schedule.

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent cytomegalovirus infection.

Data show people with pulmonary arterial hypertension tend to stay in the hospital longer and have higher medical bills.

The drug’s developer, Cereno Scientific, believes its oral therapy could have a major impact on the pulmonary arterial hypertension market.

Few question the benefits of adopting a universal healthcare data exchange standard but achieving that goal will prove to be difficult.

New research could pave the way toward a new method of pulmonary arterial hypertension diagnosis and monitoring.

Women are more likely to have pulmonary arterial hypertension, but men tend to have worse outcomes and worse treatment responses.

The technology to leverage healthcare data exists. The problem, according to Highlander Health, is outdated structures and silos.

Gossamer also reported encouraging preclinical data related to the combination of seralutinib and Winrevair.

The move means the pulmonary arterial hypertension drug is now available in the United States, Europe, and the United Kingdom.

A pooled analysis of two prior trials suggests patients with a variety of cardiac index scores saw improvement when Winrevair was added to background therapy.

All participants will now have access to the drug through an open-label extension study.

Vitamin D receptor has antiproliferative effects in pulmonary arterial hypertension, but receptor expression is downregulated in the pulmonary artery smooth muscle cells of patients.

New data suggests clinicians may be slower to recognize sepsis in people with pulmonary arterial hypertension.

Patients with pulmonary arterial hypertension often experienced delays in obtaining a correct diagnosis.