The American Journal of Managed Care

Improving SMA HRQOL Must Address Patient and Caregiver Well-being

Data from 2 prospective studies showed correlations between impaired patient health-related quality of life (HRQOL) and disease severity, as well as correlations between the health of caregivers and ...
The American Journal of Managed Care

The Future of Disease-Modifying Therapies for Patients With SMA

Significant unmet needs in SMA management include therapy access, fatigue, reproductive health, and emerging phenotypes in older patients. Intrathecal gene therapy offers potential for older SMA ...
Yahoo

Novartis wins approval to use SMA gene therapy in older patients

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. The Food and Drug Administration on Monday approved a ...
Nasdaq

Zolgensma® data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival, increased motor function ...

Basel, October 1, 2020 – Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma® (onasemnogene abeparvovec) that demonstrated patients ...
Healio

Biogen to launch study of higher Spinraza dose in patients with later onset SMA

Please provide your email address to receive an email when new articles are posted on . Biogen announced it intends to initiate a global phase 3b study to investigate clinical outcomes and safety of a ...
Morningstar

UAE’s Medcare Treats First International SMA Patient With Revolutionary Intrathecal Gene Therapy

Medcare Women & Children Hospital has successfully administered a pioneering intrathecal gene therapy for Spinal Muscular Atrophy (SMA) to Hulus, a three-year-old patient from Turkey. This milestone ...
FiercePharma

Roche touts Evrysdi win in previously treated SMA patients, turning up heat in rivalry with Biogen and Novartis

Armed with new data from what Genentech is touting as one of the biggest and most diverse studies of previously treated spinal muscular atrophy (SMA) patients, the Roche unit is padding its case for ...
Business Wire

New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a ...
Hosted on MSN

Patient health body opposes move to bar generic SMA drug

The move by Swiss multi-national, Roche (F. Hoffmann-La Roche AG) , seeking permanent injunction to prevent the introduction of a generic drug for Spinal Muscular Atrophy (SMA) jeopardises public ...