An ongoing open-label extension study suggests that the therapy's benefits may extend through at least 52 weeks. Deupirfenidone (LYT-100), a next-generation antifibrotic therapy, demonstrated a ...

Cardiac scintigraphy had higher sensitivity and specificity across different radiotracers in diagnosing cardiac amyloidosis compared to cardiac magnetic resonance imaging. Cardiac scintigraphy, ...

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), ...

The vast capabilities of artificial intelligence mean that it can applied to various domains of medicine, including in the rare disease space. To much of the public, artificial intelligence (AI) may ...

NMOSD is a rare autoimmune disorder characterized by inflammation and demyelination of nerves in the central nervous system (CNS), particularly the optic nerves and spinal cord. It typically manifests ...

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 ...

In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...

Both a higher Charlson Comorbidity Index score and older age showed a significant prognostic impact on all-cause mortality. Chronic lymphocytic leukemia (CLL) progression was the leading cause of ...

US and European regulators could approve up to 17 gene therapies this year, with a top official at the US Food and Drug Administration (FDA) predicting that 2024 will be a “breakout year” in ...

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...

Obeticholic acid, seladelpar, and elafibranor all significantly increased the biochemical response rate. Elafibranor seems to be slightly more effective than seladelpar as a second-line therapy in ...

Extreme, unrelenting fatigue has been my most intense symptom of primary biliary cholangitis (PBC) for many years now. It controls my life in many ways; it is a kind of tiredness that you cannot just ...