Novartis announces positive FDA Advisory Committee recommendation for use of Entresto® to treat patients with HFpEF
December 15, 2020 19:17 ET
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Novartis International AG
The Committee voted 12 to 1 that the data presented support the use of Entresto in treatment of patients with heart failure with preserved ejection fraction (HFpEF)Potential Q1 2021 sNDA approval...
Axiom will be Presenting and Exhibiting at Clinical Trials in Rare Diseases 2020: A Virtual Event
December 02, 2020 11:36 ET
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Axiom Real-Time Metrics Inc
TORONTO, Dec. 02, 2020 (GLOBE NEWSWIRE) -- Axiom Real-Time Metrics (“Axiom”), premier provider of unified eClinical solutions and services, is a presenting sponsor and exhibitor at Clinical Trials...
AskBio and Selecta Biosciences Receive Orphan Drug Designation for MMA-101 to Treat Methylmalonic Acidemia
November 19, 2020 08:00 ET
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Asklepios BioPharmaceutical, Inc.
RESEARCH TRIANGLE PARK, N.C. and WATERTOWN, Mass., Nov. 19, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio) and Selecta Biosciences, Inc. (NASDAQ: SELB) today announced that the...
Selecta Biosciences and AskBio Receive FDA Rare Pediatric Disease Designation for their Gene Therapy for Methylmalonic Acidemia
October 20, 2020 08:00 ET
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Asklepios BioPharmaceutical, Inc.
WATERTOWN, Mass., and Research Triangle Park, N.C., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced the...
Zolgensma® data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival, increased motor function and milestone achievement
October 01, 2020 01:15 ET
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Novartis International AG
Nearly two-thirds of patients (65.6%) in STR1VE-EU have already achieved developmental motor milestones not observed in the natural history of SMA Type 1 at a mean duration of follow-up of 10.6...
Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
August 29, 2020 06:30 ET
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Novartis International AG
Oral, investigational complement factor B inhibitor LNP023 substantially improved hematological response as add-on therapy to eculizumab Seven of ten patients discontinued eculizumab and remained on...
AskBio Appoints Michael Kranda as SVP Therapeutic Practice Leader for Its Growing CNS Portfolio
August 06, 2020 08:00 ET
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Asklepios BioPharmaceutical, Inc.
RESEARCH TRIANGLE PARK, N.C., Aug. 06, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a leading, clinical-stage adeno-associated virus (AAV) gene therapy company, today...
Allsup Joins NORD To Address COVID-19 Concerns
July 29, 2020 13:34 ET
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Allsup
Belleville, Illinois, July 29, 2020 (GLOBE NEWSWIRE) -- Patients and families affected by rare diseases are gravely concerned about COVID-19 and its impact on their health and financial well-being,...
AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases
April 22, 2020 08:05 ET
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Asklepios BioPharmaceutical, Inc.
RESEARCH TRIANGLE PARK, N.C. and PARIS, April 22, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a leading, clinical-stage adeno-associated virus (AAV) gene therapy company,...
AveXis receives positive CHMP opinion for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)
March 27, 2020 08:59 ET
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Novartis International AG
Zolgensma® (onasemnogene abeparvovec), a one-time administered gene therapy, has been recommended for European Commission (EC) conditional approval for patients with spinal muscular atrophy (SMA) and...