Author Archives: Özge Özkaya, PhD

Approximately one in six children and young people with spinal muscular atrophy (SMA) have abnormal sleep, according to a study published in the scientific journal Sleep Medicine. An additional one in six has an abnormal score on at least one sleep factor.

Access to Spinraza (nusinersen), the first ever approved therapy for the treatment of spinal muscular atrophy (SMA), will be discussed in a webinar tomorrow.

Nusinersen, an investigational drug for the treatment of spinal muscular atrophy (SMA) is acceptably safe and well tolerated, and shows encouraging clinical efficacy according to a study published in the leading medical journal The Lancet.

PTC Therapeutics announced the initiation of a Phase 2 clinical trial to test the safety, tolerability, and efficacy of the small molecule RG7916 for the treatment of children and adults with type 2 and type 3 spinal muscular atrophy (SMA).

Most people would be willing to pay to test their newborn baby for spinal muscular atrophy (SMA) even if no direct treatment is available for the disease, according to a study published in the journal Pediatric Neurology.

Developmental milestones such as sitting unaided, rolling, crawling, standing or walking are rarely, even partially achieved by infants with type 1 spinal muscular atrophy (SMA), confirms a study published in the scientific journal Neuromuscular disorders.

A team of international researchers developed a revised upper limb module (RULM) to assess arm function in people with spinal muscular atrophy (SMA), which can capture progressive muscle weakness even in the weak end of the spectrum and in young children.

Researchers from India reported the cases of two babies who were diagnosed with spinal muscular atrophy with respiratory distress syndrome (SMARD1).

Tetanus toxin may have a therapeutic role in spinal muscular atrophy (SMA) according to preliminary results published in the scientific journal Frontiers in Molecular Neuroscience.

AveXis releases the interim results of their Phase 1 clinical trial testing the gene therapy drug AVXS-101 for the treatment of SMA.