The Swiss voice in the world since 1935

Novartis unveils positive study data on Zolgensma gene therapy

Novartis new study data on Zolgensma for gene therapy treating spinal muscular atrophy (SMA).
The SMART study is reportedly the first open-label clinical trial of Zolgensma to include previously treated patients. KEYSTONE

Novartis has presented new study data on Zolgensma, its gene therapy treating spinal muscular atrophy (SMA). The data once again underscored the efficacy of this single-dose gene therapy in patients with SMA, according to a press release issued on Monday.

The results of the SMART study added to what is known about the use of Zolgensma in patients who are older (1.5 – 9.1 years) and suffer more severely than the children treated in previous clinical trials.

In almost all cases, the motoric milestones of treated patients were maintained or even improved after 52 weeks. Many of these patients had switched therapies, having previously been treated with a different disease-modifying agent.

More

The SMART study is reportedly the first open-label clinical trial of Zolgensma to include previously treated patients. Novartis announced that it will present the data at the Muscular Dystrophy Association (MDA) conference, which is taking place in the US until March 6.

Adapted from German by DeepL/dkk/amav

This news story has been written and carefully fact-checked by an external editorial team. At SWI swissinfo.ch we select the most relevant news for an international audience and use automatic translation tools such as DeepL to translate it into English. Providing you with automatically translated news gives us the time to write more in-depth articles.

If you want to know more about how we work, have a look here, and if you have feedback on this news story please write to english@swissinfo.ch.

External Content
Your subscription could not be saved. Please try again.
Almost finished… We need to confirm your email address. To complete the subscription process, please click the link in the email we just sent you.
Daily news

Get the most important news from Switzerland in your inbox.

Daily

The SBC Privacy Policy provides additional information on how your data is processed.

Popular Stories

Most Discussed

News

6.4 million loan for the Château de Gruyères voted in Fribourg

More

Gruyères castle gets public funds for renovation

This content was published on The Fribourg parliament approved a sum of CHF6.4 million to renovate the ramparts, the walkway and the towers, as well as to replace the exterior lighting.

Read more: Gruyères castle gets public funds for renovation
Increasing rental housing listings, but not placating demand

More

Swiss rental housing listings increase for the first time in three years

This content was published on For the first time in three years, the number of advertisements for rental accommodation is on the rise, but this does not dampen demand, quite the contrary. In Ticino, the average duration of an advertisement published on the main portals is 30 days.

Read more: Swiss rental housing listings increase for the first time in three years
Child vaccination rates are stagnating in many countries

More

Switzerland doing relatively well when it comes to child vaccination rates

This content was published on Progress in child immunisation has stalled. For decades, the number of children vaccinated against measles, polio and other diseases has risen. But since 2010, vaccination rates have stagnated in many countries, according to a study.

Read more: Switzerland doing relatively well when it comes to child vaccination rates
Human remains found in Blatten VS

More

Human remains found in buried Swiss village

This content was published on Human remains were found and recovered on Tuesday as part of a search operation in the buried Valais mountain village of Blatten. Formal identification is underway, according to the Valais cantonal police.

Read more: Human remains found in buried Swiss village
Lakes in Central Switzerland have the best bathing water quality

More

Lakes in Central Switzerland have best water quality for bathing

This content was published on Anyone who swims in a lake in Central Switzerland need have no fear of infection from intestinal bacteria. The water samples taken at 65 bathing sites in 13 lakes all have good to excellent bathing water quality.

Read more: Lakes in Central Switzerland have best water quality for bathing
Historic ring stolen from Basel museum

More

Historic Russian ring stolen from Basel museum

This content was published on Unknown assailants have stolen a historic ring from a Basel museum. The stolen item was a gift from Russian Tsar Alexander I to his host in Basel in 1814.

Read more: Historic Russian ring stolen from Basel museum

In compliance with the JTI standards

More: SWI swissinfo.ch certified by the Journalism Trust Initiative

You can find an overview of ongoing debates with our journalists here . Please join us!

If you want to start a conversation about a topic raised in this article or want to report factual errors, email us at english@swissinfo.ch.

SWI swissinfo.ch - a branch of Swiss Broadcasting Corporation SRG SSR

SWI swissinfo.ch - a branch of Swiss Broadcasting Corporation SRG SSR