The introduction of a disease-modifying therapy has changed the management of TK2d, making early recognition more important ...

Integrating machine learning with clinical red flags may help in the diagnosis of patients with suspected hereditary ATTR.

Italian researchers found that 62% of patients with early-stage Huntington disease (HD) lacked insight into their motor ...

Progressive pulmonary fibrosis (PPF) is undercoded and underrecognized, according to electronic health record evidence.

The US Food and Drug Administration (FDA) has accepted a supplemental New Drug Application for nebulized treprostinil for the ...

The safety profile has generally been manageable, although cytopenias, gastrointestinal symptoms, and differentiation ...

Continuing nintedanib treatment could be beneficial even in patients with PPF and IPF who would not be included in studies ...

Comprehensive care is about assessing each patient’s needs and bringing the right professionals to the table. No two cases of ...

In a new study, nearly half of individuals with NF1 had cherry angiomas, suggesting a previously unrecognized link.

Research shows that VAS-101, a transdermal curcumin gel, improves the microcirculation and tissue oxygenation in a rat model ...

A woman living with HAE recalls her worst swelling episode yet and how it has affected the way she views the good days.

Analysis of a national registry of hospital data found racial gaps in blood transfusion rates among patients hospitalized for ...