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 ...

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 ...

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 ...

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 ...

The “Abandoned, Shelved, and Rescued Therapies for Ultra-Rare Diseases” panel at the National Organization for Rare Disorders 2026 Rare Disease Scientific Symposium (Photo by Riya Ajmera) Despite ...

The authors speculated that tumor-related hypothalamic disruption may lead to CPP. In patients with CPP, 51% of OPGs were in modified Dodge classification (MDC) stage 3, the most posterior category.

Outcomes improved in patients who received nomlabofusp, while scores worsened in a matched reference cohort from the FACOMS natural history database. Larimar Therapeutics announced that the US Food ...

Ongoing multinational basket trials are enrolling patients with relapsed or refractory disease. The US Food and Drug Administration has granted Fast Track designation to gamgertamig (OM336) for the ...

Biochemical response rates were sustained through week 182 in patients with PBC treated with elafibranor, and the proportion of patients with normal alkaline phosphatase levels remained consistent.

While there is currently no cure, pharmacological therapies for progressive pulmonary fibrosis (PPF) include immunomodulatory agents that aim to reduce inflammation and antifibrotics that delay ...

Clinical practice guidelines for the diagnosis and treatment of progressive pulmonary fibrosis (PPF) in adults were published in 2022 through collaboration among the American Thoracic Society (ATS), ...

In 2020, women accounted for 75% of cases in the 26-to-35-year age group, but closer to 50% among those aged 65 years or older. Researchers have identified critical disparities in the diagnosis of and ...