Celgene Corporation and Acceleron Pharma Announce U.S. FDA Accepts Luspatercept Biologics License Application in Myelodysplastic Syndromes and Beta-Thalassemia

SUMMIT, N.J.&CAMBRIDGE, Mass.June 04, 2019 --(BUSINESS WIRE)-- Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted Celgene’s Biologics...
Source: Drugs.com - New Drug Applications - Category: Drugs & Pharmacology Source Type: news

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CONCLUSION: This study showed the frequency and pattern of chromosomal abnormalities of MDS patients in upper northern Thailand were different from other populations. MDS with isolated chromosome 5 abnormalities had clinical characteristics corresponding with patients in good prognosis risk group. However, MDS patients with chromosome 7 and complex abnormalities showed higher percentage blast of BM which high risk to progression to acute myeloid leukemia (AML). Combined CC and FISH techniques detect chromosomal abnormalities with greater frequency than when either technique is used alone.. PMID: 32212788 [PubMed - in process]
Source: Asian Pacific Journal of Cancer Prevention - Category: Cancer & Oncology Tags: Asian Pac J Cancer Prev Source Type: research
TheASXL1 mutation frequency is high in AML ‐MRC patients being its presence associated with specific characteristics, including morphological signs of dysplasia. This association raises the possible role ofASXL1 as a surrogate marker in AML ‐MRC, which could facilitate the diagnosis of patients within this group when the karyotype is normal, and especially when the assessment of multilineage dysplasia morphologically is difficult. This mutation could be used as a worst outcome marker in de novo AML‐MRC with intermediate‐risk kar yotype. AbstractAcute myeloid leukemia with myelodysplasia ‐related changes (AML...
Source: Cancer Medicine - Category: Cancer & Oncology Authors: Tags: ORIGINAL RESEARCH Source Type: research
Iron chelation therapy (ICT) with deferasirox led to longer event-free survival in iron-overloaded patients at low-to-intermediate risk of myelodysplastic syndrome (MDS) in a phase 2 study.Reuters Health Information
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Medscape Today News Source Type: news
The distinction between myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) often relies on an arbitrary marrow blast cutoff of 30% in pediatrics and 20% in adults. There is little data about the treatment of children with extramedullary myeloid malignancy that has features of both, MDS and AML. Herein, we report for the first time 2 patients MDS/AML (1 with Shwachman-Diamond syndrome and 1 with idiopathic MDS and monosomy 7) who presented with extramedullary complications, received treatment with azacitidine, achieved complete remission and subsequently underwent hematopoietic stem cell transplantation.
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Original Articles Source Type: research
Conclusion: The findings support ICT in iron-overloaded patients with low- to intermediate-1-risk MDS, with longer EFS compared with placebo and a clinically manageable safety profile. Therefore, ICT may be considered in these patients. Primary Funding Source: Novartis Pharma AG. PMID: 32203980 [PubMed - as supplied by publisher]
Source: Annals of Internal Medicine - Category: Internal Medicine Authors: Tags: Ann Intern Med Source Type: research
MONDAY, March 23, 2020 -- Iron chelation therapy (ICT) seems beneficial for iron-overloaded patients with low- or intermediate-1-risk myelodysplastic syndromes (MDS), according to a study published online March 24 in the Annals of Internal...
Source: Drugs.com - Pharma News - Category: Pharmaceuticals Source Type: news
Abstract INTRODUCTION: Hemorrhage and infection are two main causes of death in patients with myelodysplastic syndromes (MDS), and it is becoming increasingly clear that platelet dysfunction can also affect the process of hemostasis and anti-infection. The aim of this study was to evaluate activation function and immune-related function of platelets in MDS. METHODS: We included 29 MDS patients and divided them into different subgroups (low-risk group and high-risk group; untreated group and treated group; pretransfusion group and post-transfusion group) according to IPSS-R score, hypomethylating agents (HMAs)...
Source: International Journal of Laboratory Hematology - Category: Hematology Authors: Tags: Int J Lab Hematol Source Type: research
Fight Aging! publishes news and commentary relevant to the goal of ending all age-related disease, to be achieved by bringing the mechanisms of aging under the control of modern medicine. This weekly newsletter is sent to thousands of interested subscribers. To subscribe or unsubscribe from the newsletter, please visit: https://www.fightaging.org/newsletter/ Longevity Industry Consulting Services Reason, the founder of Fight Aging! and Repair Biotechnologies, offers strategic consulting services to investors, entrepreneurs, and others interested in the longevity industry and its complexities. To find out m...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Authors: Fujita M, Kamachi K, Yokoo M, Kidoguchi K, Kusaba K, Kizuka-Sano H, Yamaguchi K, Nishioka A, Yoshimura M, Kubota Y, Ando T, Kojima K, Kimura S Abstract Patients with myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) are often asymptomatic and thus can remain undiagnosed until they become symptomatic due to progression to the accelerated phase (AP) or transformation to acute leukemia (leukemic transformation; LT). We herein report the case of a previously healthy 38-year-old man who had hyperleukocytosis with dysplastic myeloid precursor cells and severe disseminated intravascular coagulation. ...
Source: Internal Medicine - Category: Internal Medicine Tags: Intern Med Source Type: research
Publication date: Available online 20 March 2020Source: Clinical Lymphoma Myeloma and LeukemiaAuthor(s): David Sallman, John Barnard, Najla H. Al Ali, Guillermo Garcia-Manero, Mikkael A. Sekeres, Amy DeZern, David P. Steensma, Gail Roboz, Elias Jabbour, Jaroslaw P. Maciejewski, Sherry Pierce, Eric Padron, Jeffrey E. Lancet, Hagop Kantarjian, Alan F. List, Rami S. Komrokji
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research
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