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 ...
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 ...
AFP

Dubai’s Medcare Hospital Becomes the World’s First to Treat an Adult Spinal Muscular Atrophy (SMA) Patient Outside the US

Medcare Royal Speciality Hospital (MRSH) in Dubai has become the world’s first hospital outside the USA, to offer a newly licensed intrathecal gene therapy - Itvisma, to adult patients with Spinal ...
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 ...
Mena FN

Dubai's Medcare Hospital Becomes The World's First To Treat An Adult Spinal Muscular Atrophy (SMA) Patient Outside The U...

Medcare Royal Speciality Hospital (MRSH) in Dubai has become the world's first hospital outside the USA, to offer a newly licensed intrathecal gene therapy - Itvisma, to adult patients with Spinal ...