Acceleron Pharma Science & Pipeline

Science & Pipeline

The publications listed below contain scientific information about investigational therapeutic candidates that are not approved for any use in any country.

Oct 1, 2019
Sotatercept
PULSAR: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Sotatercept (ACE-011) When Added to Standard of Care for the Treatment of Pulmonary Arterial Hypertension (PAH). European Respiratory Society 2019 International Congress.
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Jun 7, 2019
Sotatercept
ActIIA-Fc (Sotatercept) Reverses Pulmonary Vascular Remodeling to Attenuate Pulmonary Arterial Hypertension by Rebalancing Activin/BMP Signaling in a Preclinical Model. American Thoracic Society 2019 Annual Meeting.
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May 10, 2019
ACE-083
Results of the Dose-Escalation Portion of a Phase 2 Study of ACE-083, a Local Muscle Therapeutic, in Patients with Charcot-Marie-Tooth (CMT) Disease. American Academy of Neurology (AAN) Annual Meeting . .
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Apr 16, 2019
ACE-083
Dose Escalation Results from a Phase 2 Study of ACE-083, a Local Muscle Therapeutic, in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD). 2019 MDA Clinical & Scientific Conference. .
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Jan 10, 2019
Luspatercept
Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with beta-thalassemia. Blood. .
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Dec 2, 2018
Luspatercept
The MEDALIST Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept to Treat Patients With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) Associated Anemia With Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions. 60th ASH Annual Meeting & Exposition Plenary Session.
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Dec 1, 2018
Luspatercept
The BELIEVE Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions. 60th ASH Annual Meeting & Exposition Scientific Session.
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Nov 11, 2018
Sotatercept
Sotatercept for rebalancing BMP/TGFbeta/activin signaling in PAH. 2018 American Heart Association Scientific Session.
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Nov 11, 2018
Sotatercept
RAP-011, a Murine Ortholog of ACTRIIA-FC (Sotatercept), Improves Pulmonary Hemodynamics and Restores Right Ventricular Structure and Function in a Preclinical Model of Severe Angio-obliterative Pulmonary Arterial Hypertension. 2018 American Heart Association Scientific Session.
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Nov 10, 2018
Sotatercept
ACTRIIA-Fc Rebalances Activin/GDF and BMP9 Signaling to Attenuate Experimental Pulmonary Hypertension. 2018 American Heart Association Scientific Session.
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Oct 2, 2018
ACE-083
Preliminary Phase 2 results for ACE-083, Local Muscle Therapeutic, in Patients with CMT1 and CMTX. International Congress of the World Muscle Society . .
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Oct 2, 2018
ACE-083
Results for a Dose-Escalation Phase 2 study to Evaluate ACE-083, a Local Muscle Therapeutic, in Patients with Facioscapulohumeral Muscular Dystrophy. International Congress of the World Muscle Society . .
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Jul 23, 2018
ACE-083
Preliminary Ph 2 Results for ACE-083, Local Muscle Therapeutic, in Patients with CMT1 and CMTX. Peripheral Nerve Society . .
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Jul 23, 2018
ACE-083
Preliminary Phase 2 Results for ACE-083, Local Muscle Therapeutic, in Patients with CMT1 and CMTX. Peripheral Nerve Society. .
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Jun 16, 2018
Luspatercept
Luspatercept Inhibits pSmad2/3 Signaling and Promotes Erythroid Maturation Through a GATA1 Dependent Mechanism. 2018 European Hematology Association.
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Jun 16, 2018
Luspatercept
Improvements in Hemoglobin, Quality of Life, and Six-MinuteWalk Distance in Adults with β-Thalassemia Treated with Luspatercept: Long-Term Phase 2 Study. 2018 European Hematology Association.
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Jun 15, 2018
Luspatercept
Mutational and Subgroup Analyses of Lower-Risk Myelodysplastic Syndromes (MDS) Patients Treated With Luspatercept: Phase 2 PACE-MDS Study. 2018 European Hematology Association.
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Jun 4, 2018
Luspatercept
Erythropoietic cellular analyses in luspatercept-treated lower-risk myelodysplastic syndromes (MDS): Phase 2 PACE-MDS study. 2018 American Society of Clinical Oncology. .
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Jun 4, 2018
Luspatercept
A Phase 2, Multicenter, Open-Label Study of the Safety and Efficacy of Luspatercept in Subjects With Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis and Anemia With or Without RBC Transfusion Dependence. 2018 American Society of Clinical Oncology . .
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Apr 26, 2018
ACE-083
Preliminary Results from a Phase 2 Study to Evaluate ACE-083, a Local Muscle Therapeutic, in Patients with Facioscapulohumeral Muscular Dystrophy. 2018 American Academy of Neurology . .
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Feb 28, 2018
ACE-083
. Locally Acting ACE-083 Increases Muscle Volume in Healthy Volunteers. Muscle & Nerve. .
Dec 10, 2017
Luspatercept
Mutational Profile and Analysis of Lower-Risk Myelodysplastic Syndromes (MDS) Patients Treated with Luspatercept: Phase 2 PACE-MDS Study. American Society of Hematology . .
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Nov 14, 2017
Sotatercept
ACTRIIA-Fc rebalances BMP and activin/TGF-beta signaling to attenuate experimental pulmonary hypertension. American Heart Association 2017 Scientific Sessions. .
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Sep 1, 2017
Luspatercept
Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study. The Lancet Oncology 2017. .
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Jul 11, 2017
ACE-083
An Ongoing Phase 2 Study Evaluating the Safety, Efficacy, and Pharmacokinetics of ACE-083 in Patients with CMT1 and CMTX. 2017 Peripheral Nerve Society Annual Meeting. .
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Jul 11, 2017
ACE-083
ACE-083, A Locally-Acting GDF/Activin Ligand Trap, Augments Dorsiflexor Muscle Function in a Murine Model of Charcot-Marie-Tooth (CMT) Disease. 2017 Peripheral Nerve Society Annual Meeting. .
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Jul 9, 2017
ACE-083
Preliminary Results For Charcot-Marie-Tooth Patient-Reported Survey. 2017 Peripheral Nerve Society Annual Meeting. .
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Jun 23, 2017
Luspatercept
Luspatercept Increases Hemoglobin and Decreases Transfusion Burden in Adults With Beta-Thalassemia. European Hematology Association Congress. .
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Jun 23, 2017
Luspatercept
Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients With Lower-Risk Myelodysplastic Syndromes (MDS): Long-Term Results From the Phase 2 PACE-MDS Study. European Hematology Association Congress. .
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May 6, 2017
Luspatercept
Luspatercept Response in New Subpopulations of Patients With Lower-Risk Myelodysplastic Syndromes (MDS): Update of the PACE Study. MDS Symposium 2017. .
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Dec 5, 2016
Luspatercept
Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low-Intermediate Risk Myelodysplastic Syndromes (MDS): Long-Term Results from Phase 2 PACE-MDS Study. ASH 2016. .
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Dec 5, 2016
Luspatercept
Luspatercept Increases Hemoglobin, Decreases Transfusion Burden, and Improves Patient-Reported Outcomes in Adults with Beta-Thalassemia. ASH 2016. .
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Dec 4, 2016
Luspatercept
Pharmacokinetics and Exposure–Response of Luspatercept in Patients With Beta-Thalassemia: Preliminary Results From Phase 2 Studies. .
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Dec 4, 2016
Luspatercept
Pharmacokinetics and Exposure–Response of Luspatercept in Patients With Anemia Due to Low- or Intermediate-1-Risk Myelodysplastic Syndromes (MDS): Preliminary Results From Phase 2 Studies. ASH 2016. .
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Nov 10, 2016
ACE-083
A Phase 2 study to evaluate ACE-083, a local muscle therapeutic, in patients with FSHD. FSH Society 2016 Research Connect Conference. .
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Oct 6, 2016
ACE-083
A Phase 1 Healthy Volunteer Study of ACE-083, a Novel, Locally-Acting Muscle Agent. Patient-Centered Charcot-Marie-Tooth Summit. .
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Sep 9, 2016
ACE-083
A Phase 1 Healthy Volunteer Study of ACE-083, a Novel, Locally-Acting Muscle Agent. 6th International CMTR Consortium Meeting. .
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Jul 8, 2016
ACE-083
ACE-083, A Locally-Acting Muscle Agent, Increases Muscle Volume In Healthy Volunteers. 14th International Congress on Neuromuscular Diseases. .
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Jun 12, 2016
Luspatercept
Luspatercept Decreases Transfusion Burden and Liver Iron Concentration in Regularly Transfused Adults with Beta-Thalassemia. 21st Congress of the European Hematology Association. .
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Jun 11, 2016
Luspatercept
Luspatercept Increases Hemoglobin, Reduces Liver Iron Concentration and Improves Quality of Life in Non-Transfusion Dependent Adults with Beta-Thalassemia. Congress of the European Hematology Association. .
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Jun 10, 2016
Luspatercept
Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low- Intermediate Risk Myelodysplastic Syndromes (MDS): Long-Term Results from Phase 2 PACE‐MDS Study. 21st Congress of the European Hematology Association. .
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Jun 10, 2016
Luspatercept
RAP-536 (Murine Analog of ACE-536/Luspatercept) Inhibits SMAD2/3 Signaling and Promotes Erythroid Differentiation by Restoring GATA-1 Function in a Murine Model of Beta-Thalassemia. 21st Congress of the European Hematology Association. .
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Apr 2, 2016
ACE-083
ACE-083, a Locally-Acting TGF-Beta Superfamily Ligand Trap, Increases Muscle Volume of Targeted Muscle: Preliminary Results from a Phase 1 Dose Escalation Study in Healthy Volunteers. ENDO 2016. .
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Mar 21, 2016
ACE-083
ACE-083, a locally-acting TGF-beta superfamily ligand trap, increases muscle volume of targeted muscle: Preliminary results from a Phase 1 dose escalation study in healthy volunteers. 2016 MDA Clinical Conference. .
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Mar 21, 2016
ACE-083
ACE-083, a Locally-Acting TGF-β Superfamily Ligand Trap, Increases Muscle Mass and Strength in a Mouse Model of ALS. 2016 MDA Clinical Conference. .
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Dec 10, 2015
ACE-083
Phase 1 dose escalation study of ACE-083 in healthy volunteers: Preliminary results for a locally acting muscle therapeutic. The Society on Sarcopenia, Cachexia and Wasting Disorders (SCWD) Conference. .
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Dec 7, 2015
Sotatercept
Phase 1 Dose-Escalation Study of Sotatercept (ACE-011) in Combination with Lenalidomide and Dexamethasone in Patients with Relapsed and/or Refractory Multiple Myeloma. The 57th Annual American Society of Hematology. .
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Dec 7, 2015
ACE-1332
TGFβ1 Antagonist Inhibits Fibrosis in a Murine Model of Myelofibrosis. The 57th Annual American Society of Hematology. .
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Dec 7, 2015
Luspatercept
RAP-536 (Murine ACE-536/Luspatercept) Inhibits Smad2/3 Signaling and Promotes Erythroid Differentiation by Restoring GATA-1 Function in Murine β-Thalassemia. The 57th Annual American Society of Hematology. .
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Dec 7, 2015
Luspatercept
Luspatercept (ACE-536) Reduces Disease Burden, Including Anemia, Iron Overload, and Leg Ulcers, in Adults with Beta-Thalassemia: Results from a Phase 2 Study. The 57th Annual American Society of Hematology. .
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Dec 6, 2015
Luspatercept
Biomarkers of Ineffective Erythropoiesis Predict Response to Luspatercept in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS): Final Results from the Phase 2 PACE-MDS Study. The 57th Annual American Society of Hematology. .
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Dec 5, 2015
Luspatercept
Luspatercept Treatment Leads to Long Term Increases in Hemoglobin and Reductions in Transfusion Burden in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS): Preliminary Results from the Phase 2 PACE-MDS Extension Study. The 57th Annual American Society of Hematology. .
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Nov 6, 2015
Sotatercept
Safety and Hemoglobin Effect of Sotatercept, Administered Intravenously and Subcutaneously, for Maintenance of Hemoglobin in Hemodialysis Subjects: Interim Analysis of a Phase 2 Study. The 48th Annual American Society of Nephrology Kidney Week. .
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Nov 5, 2015
Sotatercept
Sotatercept Improves Anemia, Vascular Calcification, and Bone Loss in Patients With End-Stage Kidney Disease on Hemodialysis. American Society of Nephrology Kidney Week 2015. .
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Oct 5, 2015
ACE-083
Poster Presentation – A phase 1 dose escalation study of ACE-083, a locally-acting muscle therapeutic, in healthy volunteers. FSHD International Research Consortium. .
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Oct 1, 2015
ACE-2494
ACE-2494, a Novel GDF Ligand Trap, Increases Muscle Mass Upon Systemic Administration in Mice. 20th International Congress of the World Muscle Society. .
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Oct 1, 2015
ACE-083
ACE-083 Increases Muscle Hypertrophy and Strength in C57BL/6 Mice. 20th International Congress of the World Muscle Society. .
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Sep 14, 2015
Sotatercept
Sotatercept (ACE-011) for the Treatment of Chemotherapy-Induced Anemia in Patients with Metastatic Breast Cancer or Advanced or Metastatic Solid Tumors Treated with Platinum-Based Chemotherapeutic Regimens: Results from Two Phase 2 Studies. Supportive Care in Cancer 2015. .
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Jun 13, 2015
Luspatercept
Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low or Intermediate-1 Risk MDS: Preliminary Results from the Phase 2 PACE-MDS Study. 20th Congress of the European Hematology Association. .
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Jun 12, 2015
Luspatercept
Luspatercept Increases Hemoglobin and Decreases Transfusion Burden and Liver Iron Concentration in Adults with Beta-Thalassemia: Preliminary Results from a Phase 2 Study. 20th Congress of the European Hematology Association. .
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May 30, 2015
Sotatercept
Quantitative Computed Tomography Results for Bone Mass and Abdominal Aortic Vascular Calcification in Hemodialysis Subjects Treated With Escalating Dose Levels of Sotatercept: Interim Analysis of ACE-011-REN-001. 52nd ERA-EDTA Congress. .
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May 29, 2015
Sotatercept
The Role of Activin Signaling in the Pathogenesis of Renal Osteodystrophy of CKD-MBD. 52nd ERA-EDTA Congress. .
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May 29, 2015
Sotatercept
Sotatercept May 29, 2015 Long-term Effects of 3 Dose Levels of Sotatercept Compared With Placebo for Correction of Anemia in Hemodialysis Subjects: Interim Analysis of ACE-011-REN-001. 52nd ERA-EDTA Congress. .
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May 2, 2015
Luspatercept
Oral Presentation: Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS): Preliminary Results from a Phase 2 Study. 13th International Symposium on Myelodysplastic Syndromes (MDS). .
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Apr 29, 2015
Sotatercept
Oral Presentation – A Phase 2, Dose-Finding Study of Sotatercept (ACE-011) in Patients with Lower-Risk Myelodysplastic Syndromes (MDS) or Non-Proliferative Chronic Myelomonocytic Leukemia (CMML) and Anemia Requiring Transfusion. 13th International Symposium on Myelodysplastic Syndromes (MDS). .
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Dec 8, 2014
Luspatercept
Modified ActRIIB-mFc Fusion Protein Mitigates Sickling and Red Cell Pathology in a Murine Model of Sickle Cell Disease. 56th Annual American Society of Hematology Annual Meeting and Exposition. .
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Dec 8, 2014
Luspatercept
Luspatercept Increases Hemoglobin and Reduces Transfusion Burden in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes: Preliminary Results from a Phase 2 Study. 56th Annual American Society of Hematology Annual Meeting and Exposition. .
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Dec 7, 2014
Sotatercept
An Open-Label, Phase 2, Dose-Finding Study of Sotatercept in Patients With Low- or Intermediate-1-Risk Myelodysplastic Syndromes or Non-Proliferative Chronic Myelomonocytic Leukemia and Anemia Requiring Transfusion. 56th American Society of Hematology Annual Meeting and Exposition. .
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Dec 7, 2014
Luspatercept
Luspatercept Increases Hemoglobin and Decreases Transfusion Burden and Serum Ferritin in Adults with Beta-Thalassemia: Preliminary Results from a Phase 2 Study. 56th American Society of Hematology Annual Meeting and Exposition. .
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Nov 13, 2014
Sotatercept
Sotatercept: Results for Bone Mass and Vascular Calcification in Hemodialysis Subjects Treated With Escalating Doses: Interim Analysis of ACE-011-REN-001. American Society of Nephrology Kidney Week 2014. .
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Nov 13, 2014
Sotatercept
Safety and Hemoglobin Effect of the First 28-Day Dose Cycle of Sotatercept 0.7 mg/kg Compared With Lower Doses and Placebo for Correction of Anemia in Hemodialysis Subjects: Interim Analysis. American Society of Nephrology Kidney Week 2014. .
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Oct 9, 2014
ACE-083
ACE-083, a Ligand Trap for Members of the TGF-β Superfamily, Increases Muscle Mass Locally in a Mouse Model of Duchenne Muscular Dystrophy. 19th International Congress of the World Muscle Society. .
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Jul 1, 2014
ACE-083
A Modified Cysteine Knot Ligand Trap of the TGF-β Superfamily, ACE-083, Increases Muscle Mass Locally in Mice. 13th International Congress on Neuromuscular Diseases. .
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Jun 19, 2014
Luspatercept
Modified Activin Receptor IIB Ligand Trap Mitigates Ineffective Erythropoiesis and Disease Complications in Murine Beta-Thalassemia. Blood 2014. vol. 123 no. 25, pp. 3864-3872, .
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Jun 14, 2014
Luspatercept
Modified ActRIIB-Fc Fusion Protein (ACE-536) Decreases Irreversible Sickle Cells in a Murine Model of Sickle Cell Disease. European Hematology Associtation 19th Congress. .
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Jun 1, 2014
Sotatercept
Long-term Effects of Sotatercept Compared With Placebo for Correction of Anemia in Hemodialysis Subjects: Interim Analysis of ACE-011-REN-001 Phase 2A Study. 51st ERA-EDTA Congress. .
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Apr 26, 2014
Luspatercept
A Phase 1 Study of ACE-536, a Regulator of Erythroid Differentiation, in Healthy Volunteers. Am. J. Hematol. .
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Apr 23, 2014
Sotatercept
Interim Analysis of ACE-011-REN-001: The First 28 Day Dose Cycle of Low and Medium Starting Doses of Sotatercept Compared to Placebo for Correction of Anemia in Hemodialysis Subjects. Poster #81. National Kidney Foundation (NKF) 2014 Spring Clinical Meeting . .
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Apr 17, 2014
Luspatercept
Targeting a New Regulator of Erythropoiesis to Alleviate Anemia. Nature Medicine. vol. 20 no. 4, pp. 335-335, .
Mar 25, 2014
Sotatercept
An Activin IIa Ligand Trap Corrects Ineffective Erythropoiesis in β-Thalassemia. Nature Medicine 2014. .
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Mar 25, 2014
Luspatercept
Transforming Growth Factor-β Superfamily Ligand Trap ACE-536 Corrects Anemia by Promoting Late-Stage Erythropoiesis. Nature Medicine 2014. .
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Mar 21, 2014
Sotatercept
Sotatercept in Patients with Osteolytic Lesions of Multiple Myeloma. British Journal of Haematology 2014. .
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Mar 18, 2014
Sotatercept
An Activin Receptor IIA Ligand Trap Promotes Erythropoiesis Resulting in a Rapid Induction of Red Blood Cells and Haemoglobin. British Journal of Haematology 2014. .
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Dec 9, 2013
Sotatercept
A Phase 2a, Open-Label, Dose-Finding Study To Determine The Safety and Tolerability Of Sotatercept (ACE-011) In Adults With Beta (β)-Thalassemia: Interim Results. Blood 2013. vol. 122, pp. 3448-3448,
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Aug 16, 2013
Sotatercept
Multiple-Dose, Safety, Pharmacokinetic, Pharmacodynamic Study of Sotatercept, a Novel Erythropoietic Agent, in Healthy Postmenopausal Women. J Clin Pharmacol. vol. 53 no. 11, pp. 1121-1130, .
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Nov 6, 2012
Sotatercept
Activin receptor antagonists for cancer-related anemia and bone disease. Expert Opinion on Investigational Drugs. vol. 22 no. 1, pp. 87-101,
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Dec 1, 2010
Sotatercept
Inhibiting activin-A signaling stimulates bone formation and prevents cancer-induced bone destruction in vivo. J. Bone Miner Res 2010. vol. 25 no. 12, pp. 2633-2646, .
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Oct 1, 2010
Sotatercept
Sotatercept, a soluble activin receptor type 2A IgG-Fc fusion protein for the treatment of anemia and bone loss. Curr Opin Mol Ther 2010. vol. 12 no. 5, pp. 586-597, .
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Apr 1, 2010
Sotatercept
A soluble activin receptor Type IIA fusion protein (ACE-011) increases bone mass via a dual anabolic-antiresorptive effect in Cynomolgus monkeys. Bone 2010. vol. 46 no. 4, pp. 1082-1088,
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Mar 16, 2010
Sotatercept
Activin A promotes multiple myeloma-induced osteolysis and is a promising target for myeloma bone disease. PNAS 2010. vol. 107 no. 11, pp. 5124-5129,
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Jan 1, 2010
Sotatercept
Treatment with a soluble receptor for activin improves bone mass and structure in the axial and appendicular skeleton of female cynomolgus macaques (Macaca fascicularis). Bone 2010. vol. 46 no. 1, pp. 64-71,
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Dec 1, 2008
Sotatercept
Single-Dose, Randomized, Double-Blind, Placebo-Controlled Study of ACE-011 (ActRIIA-IgG1) in Postmenopausal Women. J Bone Min Res 2009. vol. 24 no. 4, pp. 744-752,
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May 13, 2008
Sotatercept
A soluble activin type IIA receptor induces bone formation and improves skeletal integrity. PNAS 2008. vol. 105 no. 19, pp. 7082-7087,
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