Cure SMA Issues Booklet on Insurance Coverage for SMA Patients, Families
Cure SMA, an organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), has launched a booklet addressing…
Articles by Ana de Barros, PhD
SMA Treatment Spinraza (nusinersen) Shows Positive Results in New Data
In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the…
Good Start Genetics and Roche Join to Make Screening Tool Available to Mothers-to-be
Good Start Genetics and Roche Diagnostics have entered into a marketing agreement to sell Good Start’s GeneVu carrier screening service…
Spinraza Meets Primary Endpoint for Later-onset SMA in Phase 3 Trial
Spinraza (nusinersen) for spinal muscular atrophy (SMA) met the primary endpoint in an interim analysis from a Phase 3 clinical trial…
AveXis Announces Pivotal Trial Design of SMA Type 1 Therapy Candidate
AveXis reported that the planned pivotal clinical trial of its gene therapy candidate AVXS-101 for spinal muscular atrophy (SMA) Type…
Biogen Completes Submission of New Drug Application for SMA Therapy Nusinersen
Biogen has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration…
Therapy for Infantile-Onset SMA, Nusinersen, Shows Promise in Phase 3 Study
Biogen and Ionis Pharmaceuticals recently reported that nusinersen, an antisense drug designed to treat spinal muscular atrophy (SMA), ably…
Cure SMA to Put $2.5 Million Toward Research Efforts
Cure SMA, an organization dedicated to the treatment and cure of Spinal Muscular Atrophy (SMA), will provide $2.5 million over…
SMA Type 1 Treatment Candidate Granted FDA Breakthrough Therapy Designation
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to AVXS-101, AveXis’ lead development candidate to treat spinal…
Cure SMA Grants $304,000 to Columbia University Professor for SMA Drug Discovery
Cure SMA recently granted a $304,000 drug discovery award to Livio Pellizzoni, assistant professor of Pathology & Cell Biology at Columbia University…