Allsup Joins NORD To Address COVID-19 Concerns
July 29, 2020 13:34 ET
|
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
|
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
|
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
March 24, 2020 02:15 ET
|
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
March 24, 2020 02:13 ET
|
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)
March 19, 2020 02:15 ET
|
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
March 18, 2020 09:27 ET
|
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...
APHL Announces Newborn Screening Systems Quality Improvement Projects Second Cohort Award Recipients
February 25, 2020 11:15 ET
|
Association of Public Health Labs
Silver Spring, MD, Feb. 25, 2020 (GLOBE NEWSWIRE) -- The Association of Public Health Laboratories (APHL) is pleased to announce this year’s recipients of the Newborn Screening Systems Quality...
APHL Announces Newborn Screening Award Recipients
February 11, 2020 12:01 ET
|
Association of Public Health Labs
Silver Spring, MD, Feb. 11, 2020 (GLOBE NEWSWIRE) -- The Association of Public Health Laboratories (APHL) is pleased to announce the fall 2019 recipients of the Expansion of Spinal Muscular Atrophy...
AskBio Launches AskFirst™ Patient Advocacy Program to Educate and Support Participants in Gene Therapy Clinical Trials
January 29, 2020 10:27 ET
|
Asklepios BioPharmaceutical, Inc.
RESEARCH TRIANGLE PARK, N.C., Jan. 29, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a clinical-stage adeno-associated virus (AAV) gene therapy company, today introduced...