SMA News Today

May 16, 2022May 16, 2022

From Actor to Attorney, Woman’s ‘Work Ethic Outshines My Disability’

SMA News Today

From being a child actor to a lawyer in the entertainment industry, Alexa Dectis, who has relied on a power wheelchair to get around since she was 2, has gone ... Read more

May 13, 2022May 13, 2022

Spinraza Stabilizes Motor Function for Adults With SMA, Study Reports

SMA News Today

Treatment with Spinraza (nusinersen) modestly improved muscle strength and stabilized motor function for adults with spinal muscular atrophy (SMA) types 2 or 3, according to a study from Israel. The ... Read more

May 11, 2022May 11, 2022

New Gene Therapy Shows Potential for Safety, Efficacy in Mouse Model

SMA News Today

A second-generation investigational gene therapy for spinal muscular atrophy (SMA) showed greater safety and efficacy than an earlier version, and it may be more effective than the currently approved gene ... Read more

May 9, 2022May 9, 2022

Parents of SMA Children Put Health as Top Priority at COVID-19’s Onset

COVID-19 News, SMA News Today

For parents, protecting the health and well-being of a child with spinal muscular atrophy (SMA) was a top priority when the COVID-19 pandemic first hit the Netherlands in early 2020, ... Read more

May 6, 2022May 6, 2022

Disease Severity May Be Impacted by SMN Protein in Rare SMA Cases

SMA News Today

The stability and self-interacting ability of SMN, the protein that is abnormally low in people with spinal muscular atrophy (SMA), may impact disease severity in rare cases, a small study ... Read more

May 4, 2022May 4, 2022

Type 1 Children on Evrysdi Continue to Hit Milestones After 3 Years

SMA News Today

Children with spinal muscular atrophy (SMA) type 1 who started treatment with Evrysdi (risdiplam) in the first months of life are continuing to gain motor skills after three years of treatment, ... Read more

May 2, 2022May 2, 2022

Girl, 16, Becomes 1st SMA Patient to Undergo Hip Replacement Surgery

SMA News Today

A surgeon at UC Davis Health has successfully performed the first hip replacement in a person — in this case, a 16-year-old girl — with spinal muscular atrophy (SMA). Before ... Read more

April 29, 2022April 29, 2022

#AAN2022 – Spinraza Showing Safety in Children With Needs After Zolgensma

SMA News Today

Treatment with Spinraza (nusinersen) appears safe in infants and children with spinal muscular atrophy (SMA) who responded poorly to the gene therapy Zolgensma, early safety data from the first nine RESPOND trial ... Read more

April 29, 2022April 29, 2022

Organizations Rally to Help Ukrainian Rare Disease Patients

SMA News Today

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ... Read more

April 27, 2022April 27, 2022

#AAN2022 – Apitegromab Prompts Motor Improvements in TOPAZ Trial

COVID-19 News, SMA News Today

Treatment with the experimental muscle-directed therapy apitegromab improved motor function for many of the children and young adults with types 2 and 3 spinal muscular atrophy (SMA) after one year ... Read more

April 25, 2022April 25, 2022

SMN-deficient Brain Immune Cells Found Prone to Promote Inflammation

SMA News Today

Loss of the survival motor neuron (SMN) protein changes the function of a type of brain immune cell called microglia, making them more prone to promote inflammation and disease progression ... Read more

April 22, 2022April 22, 2022

Combining Therapies May Improve Efficacy in SMA: Mouse Model

SMA News Today

A combined spinal muscular atrophy (SMA) therapy — one incorporating two treatments targeting different disease processes related to the SMN protein — showed improved efficacy over single treatments in a ... Read more

April 21, 2022April 21, 2022

Europe Awaits Proposed New Framework for Sharing Health Data

SMA News Today

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health ... Read more

April 20, 2022April 20, 2022

Researchers Share Story of Patient Who Inspired Them

SMA News Today

Researchers in Hong Kong have shared a case report about how they were inspired and humbled by a teenage girl with spinal muscular atrophy (SMA) who got through a difficult ... Read more

April 18, 2022April 18, 2022

20 Liberty Trikes To Be Donated to Children with SMA, Other Diseases

SMA News Today

Electric Bike Technologies (EBT) will donate 20 of its Liberty Trikes this year to help children with limited abilities get exercise and enjoy bike rides with their family and friends. ... Read more