Axiom will be Presenting and Exhibiting at Clinical Trials in Rare Diseases 2020: A Virtual Event
02 déc. 2020 11h36 HE
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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
19 nov. 2020 08h00 HE
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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
20 oct. 2020 08h00 HE
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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
01 oct. 2020 01h15 HE
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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)
29 août 2020 06h30 HE
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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
06 août 2020 08h00 HE
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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
29 juil. 2020 13h34 HE
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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
22 avr. 2020 08h05 HE
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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)
27 mars 2020 08h59 HE
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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...
Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing
24 mars 2020 02h15 HE
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Novartis International AG
Interim SPR1NT data showed presymptomatic babies with SMA treated with Zolgensma® (onasemnogene abeparvovec-xioi) soon after birth achieved age-appropriate motor milestones In addition to meeting both...