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MDS patients who have failed prior HMA therapy selected as first indication to advance into Phase 2 3 mg/kg and 6 mg/kg doses selected in accordance with FDA’s Project Optimus initiative guidancePhase 2 part of the study will recruit 32 HMA-failed MDS patients for 1:1 dose randomization with possible data release after 20 patients have received more than two treatment cycles Company announcement, Inside Information TURKU, Finland and BOSTON, Nov. 06, 2023 (GLOBE NEWSWIRE) -- Faron Pharmac...

PTC Therapeutics Inc (NASDAQ: PTCT) shared interim data from the 12-week portion of the PIVOT-HD Phase 2 study of PTC518 in Huntington's disease (HD) patients. The study demonstrated dose-dependent lowering of Huntingtin (HTT) protein levels in peripheral blood cells, reaching a mean 30% reduction in mutant HTT levels at the 10mg dose level. In addition, PTC518 exposure in the cerebrospinal fluid (CSF) was consistent with or higher than plasma unbound drug levels. PTC518 treatment was also ...

-- WU-CART-007 was well-tolerated in patients with R/R T-ALL/LBL, with low incidence of high-grade CRS and immune-related adverse events -- -- WU-CART-007 demonstrated preliminary evidence of anti-leukemic

- FS118 is Well-Tolerated Across all Dose Levels with No Serious Adverse Events Related to FS118 Therapy - Novel mechanism showing Increased Peripheral Immune Cells and LAG-3 shedding - 54.8%

Fatigue 17 3 12 2 Lipase increased 17 3 17 2 Anaemia 16 5 2 - Amylase increased 15 1 13 1 Nausea 12 1 7 - Vomiting 12 3 4 - Oedema peripheral 11 - 9 - Abdominal pain 10 2 1 -... | October 7, 2021