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 ...

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 ...

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 ...

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 ...

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 ...

Rh immunoglobulin is recommended only after small-volume (ie, ≤1 unit) RhD-positive low-titer group O whole blood or RBC transfusions in RhD-negative females of childbearing potential, with ...

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, ...

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 ...

Survival outcomes were similar with hypomethylating agents (HMAs) alone and an HMA plus venetoclax despite higher overall response rates. Investigators retrospectively evaluated 140 patients with TP53 ...

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.

A radiotherapy-sparing approach may be beneficial in vulnerable patients with NF1-associated medulloblastoma. A child with neurofibromatosis type 1 (NF1) was diagnosed with medulloblastoma, which was ...

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 ...