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

Researchers Determine What Patients, Caregivers Deem Meaningful Change in SMA Motor Function

Through the use of patient testimonials as well as analysis of data from a study of patients with spinal muscular atrophy (SMA), researchers were able to describe patient-centered “meaningful outcomes ...
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 ...
Business Wire

Exegenesis Bio to Present 9-Patient Data from a Phase 1/2 Clinical Trial of EXG001-037, a Novel rAAV Gene Therapy for Spinal Muscular Atrophy (SMA) Type 1: Improved Head ...

PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 ...
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 ...
Business Wire

Positive Phase 2 Topaz Trial Extension Data Demonstrate Sizable and Sustained Motor Function Improvement at 24 Months with Apitegromab for Non-Ambulatory Patients with Types 2 ...

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 ...
Business Insider

SMA Genetic Testing and IVF Could Save Over $3.8M per Patient vs. Novartis Gene Therapy

ORLANDO, Fla., Feb. 5, 2019 /PRNewswire-PRWeb/ -- Pharmaceutical company Novartis made headlines in 2018 with a new gene therapy to treat spinal muscular atrophy (SMA) type 1—the leading genetic cause ...
FierceBiotech

Scholar Rock's SMA drug helps weight loss patients on Zepbound retain muscle in phase 2 trial

Scholar Rock’s investigational spinal muscular atrophy drug helped patients taking Eli Lilly’s obesity blockbuster Zepbound maintain muscle mass, offering another avenue to reach a key goal for the ...