Efficacy analyses for the first 2 cohorts of the phase 1/2 studies will compare participant outcomes with those of a propensity score-matched external control group. AMT-130, an investigational gene ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

Sharon Hesterlee, PhD, who previously served as chief research officer at the MDA, has been named interim president and CEO. Donald S. Wood, PhD, has retired from the Muscular Dystrophy Association ...

The CAPTIVATE trial met its target of at least 20 responders among the first 40 participants before all 40 completed part A. Dianthus Therapeutics announced an early “go” decision for claseprubart for ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

As winter sets in, many people enjoy a hot mug of cocoa, snuggly sweaters, and outdoor activities such as walking through town to watch the lighting of the town tree or skiing with friends. There are ...

While further testing is needed in a larger sample of patients, spesolimab may be a promising option for the treatment of combined GPP and PPP. Spesolimab, an interleukin-36 (IL-36) receptor ...

In a phase 3 trial, participants taking venglustat demonstrated statistically significant improvement in neurological symptoms compared with those receiving ERT. Sanofi announced that the US Food and ...

Between 1970 and 1995, the incidence of new NHL cases increased. Between 2007 and 2016, new cases declined by approximately 0.9% per year, and mortality from NHL decreased by 2.2% per year. 3 ...

Researchers advanced the therapy with an AI platform that uses machine learning models trained on patient endotyping, multiomics, biomarker, and clinical data. The US Food and Drug Administration (FDA ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Miller, MD, a professor of pediatric endocrinology at the University of Florida in Gainesville who specializes in ...