Salbutamol Improves Respiratory Muscle Strength of Children With SMA, Study Reports
A year’s worth of salbutamol improved the breathing function and respiratory muscle strength of children with spinal muscular atrophy, according…
Articles by Joana Fernandes, PhD
Education Is Essential to Success of SMA Carrier Screening Programs, U.K. Study Finds
Families living with spinal muscular atrophy (SMA), like most people, favor screening for genetic disorders. Yet those who lack experience…
Cytokinetics Shares Baseline Characteristics of the First SMA Patients Enrolled in Phase 2 Trial Testing CK-2127107
Cytokinetics revealed preliminary data on the characteristics of patients with spinal muscular atrophy (SMA) screened for a Phase 2…
RG7916 Targets Underlying Genetic Cause of Type 2 and 3 SMA, Preliminary Trial Results Show
Preliminary results from a phase 2 trial confirm that the investigational drug RG7916 targets the underlying genetic cause of…
Cure SMA Pursues More Education and Better Clinical Trial Access, Capacity
Cure SMA announced the start of Phase 2 of its SMA Industry Collaboration, aiming to advance the development of…
Blocking Protein Receptor May Prevent Motor Neuron Death, SMA Study Reveals
Blocking a protein receptor called m2R may prevent motor neuron death, leading to new therapies for spinal muscular atrophy (SMA), according…
Cure SMA to Host May 4 Webinar on Spinraza Access, Insurance Coverage and Trial Results
The patient advocacy group Cure SMA will host a webinar May 4 on access to Spinraza (nusinursen), the first drug ever to…
AveXis’ AVXS-101 Shows Promise in Treating Infants with SMA Type 1
AVXS-101 may become a valuable gene therapy option for babies with spinal muscular atrophy (SMA) type 1, according to the results…
Invitae Rolls Out New Genetic Test to Diagnose SMA
San Francisco-based Invitae has developed a genetic test to diagnose spinal muscular atrophy (SMA), the company announced at the March 21-25 …
Most SMA Moms Reported Positive Experiences During Pregnancy and Delivery, Study Reports
Researchers conducted a questionnaire-based study to learn more about pregnancy and delivery in women with spinal muscular atrophy (SMA), knowing…