SMA News Today

July 28, 2021July 28, 2021

Spinraza Improves Lung Function in SMA Type 2 Child, Report Says

SMA News Today

Spinraza (nusinersen), a treatment for spinal muscular atrophy (SMA) that helps improve motor function, was found to also improve lung function in a 10-year-old girl with advanced SMA type 2, ... Read more

July 27, 2021July 27, 2021

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

SMA News Today

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other ... Read more

July 26, 2021July 26, 2021

Novartis Stopping Work on Branaplam as Oral SMA Therapy

SMA News Today

Novartis is stopping the clinical development of branaplam (LMI070), its experimental oral treatment for spinal muscular atrophy (SMA) being evaluated in a Phase 1/2 clinical trial. According to the company’s announcement, ... Read more

July 23, 2021July 23, 2021

Early Work Supports Apitegromab’s Safety as SMA Muscle Therapy

SMA News Today

A series of toxicology studies in rats and macaque monkeys confirmed the safety and tolerability of apitegromab, an investigational spinal muscular atrophy (SMA) therapy, supporting its ongoing assessment in patient ... Read more

July 21, 2021July 21, 2021

SadBaby Crowdfunding Aims to Help SMA Families

SMA News Today

SadBaby, an organization that raises donations to support children with spinal muscular atrophy (SMA), is launching a crowdfunding platform. The project, based on digital currency donations, intends to gather funds ... Read more

July 20, 2021July 20, 2021

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

SMA News Today

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies ... Read more

July 19, 2021July 19, 2021

‘Wearable Cyborg’ Helps Improve Walking Ability, Muscle Strength

COVID-19 News, SMA News Today

A wearable cybernetic leg significantly improved the gait and ability to perform walking exercises of people with neuromuscular disorders, such as spinal muscular atrophy (SMA), as compared to a conventional ... Read more

July 16, 2021July 16, 2021

Adult-onset SMA Form, With VRK1 Mutations, Found in 2 Hispanics

SMA News Today

Two mutations in the VRK1 gene were found in two unrelated Hispanic adults, with symptoms suggestive of an atypical form of adult-onset spinal muscular atrophy (SMA), according to a case report. While ... Read more

July 14, 2021July 14, 2021

COVID-19 Infection Turns Severe, Deadly in SMA Type 1 Child

COVID-19 News, SMA News Today

A hospitalized 3-year-old with spinal muscular atrophy (SMA) type 1 died after being infected by the virus that causes COVID-19 and having a widespread inflammatory response — unusual in infected children ... Read more

July 13, 2021July 13, 2021

Nominations Open for 2022 Eurordis Black Pearl Awards

SMA News Today

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work ... Read more

July 12, 2021July 12, 2021

SMA Treatment May Be More Effective by Altering Underlying Molecule

SMA News Today

Biochemical modifications made to antisense oligonucleotides — molecules that are the basis of an existing therapy for spinal muscular atrophy (SMA) — may improve this and similar treatments, new research suggests. ... Read more

July 9, 2021July 9, 2021

Spinraza Improves Motor Function in SMA Type 3 Children, Too

SMA News Today

About one year of treatment with Spinraza (nusinersen) effectively prevents or reverses motor function decline in children and adults with spinal muscular atrophy (SMA) type 3, according to real-world data from an international ... Read more

July 7, 2021July 7, 2021

Stem Cell Therapy May Benefit Some Type 1 Infants

SMA News Today

Treatment with human fat tissue-derived mesenchymal stem cells (MSCs) may improve motor nerve cell health and survival in infants with spinal muscular atrophy (SMA) type 1, according to data from a small ... Read more

July 2, 2021July 2, 2021

People in UK Urged to Sign Petition Adding SMA to Newborn Screening

SMA News Today

The UK SMA Newborn Screening Alliance is calling on people to take action and sign a petition requesting that spinal muscular atrophy (SMA) to be added to the U.K.’s National ... Read more

June 30, 2021June 30, 2021

US Pediatricians Often Overlook Steps in Diagnosing SMA, Surveys Show

SMA News Today

Many U.S. pediatricians fail to use developmental screening tools and are not familiar with the diagnostic requirements for spinal muscular atrophy (SMA), a study drawing on results of two surveys ... Read more