SMA News Today

April 12, 2021April 12, 2021

Immune Response to Zolgensma Can Be Common in Older SMA Children

SMA News Today

Abnormal increases in liver enzymes levels — suggestive of liver damage — and drops in platelet counts are common among spinal muscular atrophy (SMA) patients over 8 months old following treatment ... Read more

April 9, 2021April 9, 2021

Apitegromab Over 1 Year Bolsters Motor Abilities of Types 2 and 3: TOPAZ Trial

COVID-19 News, SMA News Today

Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), safely and effectively improved or stabilized motor function in children and young adults with types 2 and 3 disease over one year, top-line ... Read more

April 7, 2021April 7, 2021

European Alliance Calls for Newborn Screening

SMA News Today

The European Alliance for Newborn Screening for Spinal Muscular Atrophy is calling on all European states to introduce routine screening of all newborns for spinal muscular atrophy (SMA) by 2025. ... Read more

April 5, 2021April 5, 2021

Scientists Reveal Architecture of SMN Complex, Altered in SMA

SMA News Today

Scientists have revealed the structural architecture of the SMN complex, which is altered in people with spinal muscular atrophy (SMA) due to a deficiency in the SMN protein. Their findings showed that many ... Read more

April 2, 2021April 2, 2021

Spinal Fluid Changes With Spinraza’s Use Mild, Possibly Due to Lumbar Puncture

SMA News Today

Treatment with Spinraza (nusinersen) may cause some alterations in the cerebrospinal fluid — that which surrounds the brain and spinal cord — in adults with spinal muscular atrophy (SMA), a study ... Read more

March 31, 2021March 31, 2021

Evrysdi Approved in Europe as First Oral, At-home Treatment

SMA News Today

The European Commission has approved Evrysdi (risdiplam) as the first oral and at-home treatment for adults, children, and infants 2 months and older with nearly all types of spinal muscular atrophy (SMA). Eligible patients ... Read more

March 30, 2021March 30, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

SMA News Today

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of ... Read more

March 29, 2021March 29, 2021

Survey: Benefits of SMA Newborn Screening Outweigh Disadvantages

SMA News Today

The benefits of newborn screening (NBS) for spinal muscular atrophy (SMA) outweigh its disadvantages and challenges, according to a survey of parents and healthcare providers of screen-positive newborns in Australia. ... Read more

March 26, 2021March 26, 2021

Scholar Rock Wins US Patent for Apitegromab

SMA News Today

The United States Patent and Trademark Office has granted Scholar Rock a patent for apitegromab, with add-on and combination therapies, for treating muscle conditions such as spinal muscular atrophy (SMA). ... Read more

March 24, 2021March 24, 2021

#MDA2021 – Zolgensma Helped Pre-symptomatic Babies Achieve Age-Appropriate Motor Milestones

SMA News Today

Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Zolgensma ... Read more

March 23, 2021March 24, 2021