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FDA Boosts Orphan Drug Designations for Myelofibrosis Treatments

The rise in FDA ODD awards indicates a collective endeavor to create new myelofibrosis medications devoid of mechanisms inducing anemia. ....

Jasper Morley , National Organization For Rare Disorders , About Myelofibrosis , National Organization , Rare Disorders , Drugs Intelligence Analyst , Orphan Drugs , Blood Cancer ,

Pelabresib Plus Ruxolitinib Improves Spleen Volume Reduction in JAK Inhibitor-Naive Myelofibrosis

Treatment with the combination of pelabresib and ruxolitinib led to a statistically significant and clinically meaningful improvement in spleen volume reduction vs placebo plus ruxolitinib in patients with JAK inhibitor-naive myelofibrosis, meeting the primary end point of the phase 3 MANIFEST-2 trial. ....

New York , United States , John Mascarenhas , Rubena Mesa , Tisch Cancer Institute At Mount Sinai , European Medicines Agency , Dynamic International Prognostic Scoring System , Atrium Health Levine Cancer Center , Adult Leukemia Program , Tisch Cancer Institute , Mount Sinai , Manifest 2 Trial ,

NCCN Guidelines Update Adds Momelotinib for High-, Low-Risk Myelofibrosis

Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia.
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Women Health , Digital Health , Population Health , Nccn Guideline Updates , Jak Inhibitors , Rare Diseases ,