How Zolgensma Works and Trials Underway in Range of SMA Patients
Zolgensma (onasemnogene abeparvovec-xioi), the newly approved gene therapy to treat SMA in infants and children under 2 years old,…
Articles by José Lopes, PhD
FDA Approves Zolgensma, 1st Gene Therapy to Treat SMA in Children Up to Age 2
Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now…
#AANAM — Experts Discuss Improved Survival, Key Motor Milestones Achieved with Risdiplam in SMA Trials
Treatment with the experimental oral therapy risdiplam has been enabling patients with spinal muscular atrophy (SMA) to achieve…
#AANAM – Spinraza Showing Long-term Benefits in All SMA Types, Biogen Says in Interview
Long-term results show Spinraza (nusinersen) is of unprecedented benefit to a broad range of spinal muscular atrophy (SMA)…
#AANAM – Zolgensma Showing ‘Strong Efficacy’ Across SMA Types, AveXis Says in Interview
With the U.S. Food and Drug Administration (FDA) expected to soon decide whether Zolgensma (onasemnogene abeparvovec) can treat people with…
#AANAM – Risdiplam Shows Promise for Treating SMA Types 2 and 3, Clinical Data Shows
Risdiplam continues to show promise as a therapy for individuals with spinal muscular atrophy (SMA) types 2 and…
#AANAM – Spinraza Continues to Demonstrate Safety and Provide Improvements in SMA Patients
Treatment with Spinraza (nusinersen) results in greater improvements in motor function and continues to improve or stabilize motor…
#AANAM – Rapid Motor Function Improvement with Zolgensma in Presymptomatic Infants with SMA Types 1-3, Early Trial Results Show
Treatment of presymptomatic babies with the investigational gene therapy Zolgensma provides rapid improvement in motor function in infants with…
#AANAM – Type 2 Patients Taking Well to Zolgensma, With Motor Milestones and No Safety Concerns
Type 2 patients with spinal muscular atrophy (SMA) given the gene therapy Zolgensma via spinal canal injection are showing no safety…
Certain Changes in Bladder, Colon May Underlie Gastrointestinal Issues in SMA, Mouse Study Suggests
SMN protein deficiency in spinal muscular atrophy (SMA) may lead to gastrointestinal (GI) dysfunction by shrinking the smooth muscle…