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...
AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2
24 mars 2020 02h13 HE
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Novartis International AG
SMA Type 2 patients between two and five years of age who received Dose B met the primary efficacy endpoint with a remarkable mean increase of 6.0 points in HFMSE scores from baseline to month 12,...
Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma® the only gene therapy for patients with spinal muscular atrophy (SMA)
19 mars 2020 02h15 HE
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Novartis International AG
Zolgensma® (onasemnogene abeparvovec), a one-time administered gene therapy, is approved for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at...
AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome
18 mars 2020 09h27 HE
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Asklepios BioPharmaceutical, Inc.
RESEARCH TRIANGLE PARK, N.C. and CHAPEL HILL, N.C., March 18, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a leading clinical-stage adeno-associated virus (AAV) gene therapy...