SMA News Today

October 23, 2020October 23, 2020

Evrysdi Approved in Brazil for SMA, But No Indications Specified

Evrysdi (risdiplam) has been approved for the treatment of spinal muscular atrophy (SMA) in Brazil by the country’s National Health Surveillance Agency, known as ANVISA. The Brazilian approval came within ... Read more

October 21, 2020October 21, 2020

PAN Foundation Offers Financial Help for SMA Treatment

SMA News Today

People living with spinal muscular atrophy (SMA) who need financial help to pay for treatment may now apply for aid through a new patient assistance program offered by the PAN ... Read more

October 19, 2020October 19, 2020

Risdiplam, Oral SMA Treatment, Up for Approval in Japan

SMA News Today

Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for spinal muscular atrophy (SMA) patients in Japan. A new drug application seeking approval, submitted ... Read more

October 16, 2020October 16, 2020

Adults With SMA Who Sought Specialty Care After Spinraza Approval May Have More Severe Disease

SMA News Today

Adults with spinal muscular atrophy (SMA) who began being seen at specialty centers after the approval of Spinraza (nusinersen) may have more severe symptoms than adults who have been followed regularly ... Read more

October 14, 2020October 14, 2020

‘Think 3 at 3 Months’ Drive Seeks to Raise Awareness of Infant Movement Milestones

SMA News Today

A new campaign is seeking to raise awareness of key motor development milestones for infants that, if missed, could mean spinal muscular atrophy (SMA) or other neuromuscular conditions. It’s been ... Read more

October 12, 2020October 12, 2020

Gene Therapy Reduces Disease Severity in SMA Mouse Model, Study Shows

SMA News Today

Gene therapy that increased the production of a protein called DOK7 in a spinal muscular atrophy (SMA) mouse model was found to improve the neuromuscular junction structure, reduce muscle fiber ... Read more

October 9, 2020October 9, 2020

SMA Type 3 Adults May Have Issues Regulating Component of Cell’s ‘Skeleton,’ Study Suggests

SMA News Today

Adults with spinal muscular atrophy (SMA) type 3 have significantly lower levels of several molecules that help regulate actin, one of the major components of the cell’s cytoskeleton, according to ... Read more

October 7, 2020October 7, 2020

EU Approves EONIS System to Screen Newborns for SMA, Other Conditions

SMA News Today

The EONIS system, a screening assay capable of simultaneously testing newborns for three genetic conditions, including spinal muscular atrophy (SMA), has received a CE marking from the European Commission for ... Read more

October 5, 2020October 5, 2020

4-aminopyridine Fails to Improve Muscle Function in SMA Type 3 Patients, Small Trial Finds

SMA News Today

Treatment with the molecule 4-aminopyridine (4-AP) failed to improve the motor function, endurance, and muscle strength of spinal muscular atrophy (SMA) type 3 patients who are able to walk, according ... Read more

October 2, 2020October 2, 2020

SMA Type 1 Infants Treated With Zolgensma Achieving Milestones, Data Show

SMA News Today

Infants with spinal muscular atrophy (SMA) type 1 who received a single infusion of Zolgensma continue to achieve developmental milestones and show improvements in motor function, according to interim data from the ... Read more

September 29, 2020September 30, 2020

Infants Receiving Evrysdi Continue to Improve and Achieve Motor Milestones, Phase 2/3 Trial Data Show

SMA News Today

Infants with spinal muscular atrophy (SMA) type 1 receiving the therapeutic dose of Evrysdi (risdiplam) continue to improve and achieve motor milestones, according to two-year data from the first part ... Read more

September 24, 2020September 24, 2020

Novartis to Open, at FDA Request, New Trial of Zolgensma for Older Patients

SMA News Today

Novartis will open a new clinical trial investigating Zolgensma, given as a one-time injection directly into the spinal canal, after the U.S. Food and Drug Administration (FDA) recommended a “pivotal confirmatory ... Read more

September 23, 2020September 23, 2020

Spinraza Leads to Motor Gains Over Time in SMA Type 3 Adults, Real-Life Study Finds

SMA News Today

About one year of treatment with Spinraza (nusinersen) gradually and significantly improves motor function in adults with spinal muscular atrophy (SMA) type 3 — especially those unable to walk without ... Read more

September 21, 2020September 21, 2020

Evrysdi Open to Eligible Patients in UK Through Early Access Program

SMA News Today

Evrysdi (risdiplam), Roche’s oral therapy for spinal muscular atrophy (SMA), will be available to select patients in the U.K. before its potential regulatory approval through an Early Access to Medicines ... Read more

September 18, 2020September 18, 2020

GridPad Trilogy Communication Device Aids People With Speech and Motor Disorders

SMA News Today

Control Bionics has announced the launch of the GridPad Trilogy, an augmentative and alternative communication (AAC) device intended to aid people living with a range of conditions that affect movement and ... Read more