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.

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 ...

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 ...

When tested on Investigational New Drug products (n=7), both complement-dependent hemolysis and monocyte monolayer assays effectively detected elevated hemolytic activity. The Food and Drug ...

In diagnosed cases of cutaneous mastocytosis, investigations to rule out systemic mastocytosis may be appropriate. A young child with a small chest lump and anemia was found to have cutaneous ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

Long-term safety was comparable to standard of care, with no new safety signals identified over 52 weeks. CAM2029, a long-acting octreotide subcutaneous depot, provided sustained biochemical control, ...

Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...

Although 94% of HCPs agreed that transparent communication improves their understanding of patient preferences, only 57% of patient advocacy group representatives concurred. Physicians and patient ...

Pending approval, commercial launch is anticipated in the first half of 2027. Intellia Therapeutics announced the initiation of a rolling biologics license application (BLA) submission to the US Food ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

Regenxbio announced plans to pursue accelerated FDA approval of RGX-202 and prepare for a potential commercial launch in 2027. The pivotal portion of the trial of RGX-202, a gene therapy for Duchenne ...