I am a healthcare professional


Welcome to MEDICALLY. This website is a non-promotional international resource intended to facilitate transparent scientific exchange regarding developments in medical research and disease management. It is intended for healthcare professionals outside the United Kingdom (UK) and Australia. The content on this website may include scientific information about experimental or investigational compounds, indications and services that are not approved or valid in your jurisdiction. Registration status and prescribing information of medicinal products may differ between countries. Roche and Genentech do not support, endorse or recommend the unapproved use of any compound or service in your jurisdiction, including those discussed on this website.

Please refer to local product information for any medicinal products mentioned. Information available on this website does not constitute professional medical advice, and Roche and Genentech accept no responsibility for access to or use of the same. Healthcare professionals outside the US are required to register and log-in to access the full range of content available on this website.

By clicking on one of the healthcare professional buttons below, you acknowledge you have read and understood this message and that you are requesting access to MEDICALLY. If you are not a healthcare professional, please use the other links below to access information relevant to you.


We use cookies on this site to enable the site to function properly and to enhance your user experience. Cookies are files stored in your browser, which most websites use to personalize your web experience. Your information will only be used to provide information that is relevant to you. It will not be used for any other purpose. If you wish to restrict or block cookies, which are set on your device, then you can do this through your browser settings.

You can find out more about cookies by browsing our Privacy Policy.


For healthcare professionals in Another Country browse medically.roche.com.

Not a healthcare professional? For individuals outside the US, browse Roche's patient website or roche.com. For individuals in the US, browse gene.com.

External site

The link you have selected will take you away from this site to one that is not owned or controlled by Genentech, Inc. Genentech, Inc. makes no representation as to the accuracy of the information contained on sites we do not own or control. Genentech does not recommend and does not endorse the content on any third-party websites. Your use of third-party websites is at your own risk and subject to the terms and conditions of use for such sites.

Leave site
Roche and Genentech at

Cure SMA 2022

Coming soon
10:40 AM
Duration 20mins The Sequoia ballroom (Grand Californian Hotel)
RAINBOWFISH: Preliminary efficacy and safety data in risdiplam▼-treated infants with presymptomatic spinal muscular atrophy (SMA)
Richard S Finkel, Michelle A Farrar, Dmitry Vlodavets, Edmar Zanoteli, Mohammad Al-Muhaizea, Leslie Nelson, Alexandra Prufer, Laurent Servais, Yi Wang, Carolyn Fisher, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Laura Palfreeman, Renata S Scalco, Enrico Bertini, on behalf of the RAINBOWFISH Study Group​
11:00 AM
Duration 20mins The Sequoia ballroom (Grand Californian Hotel)
FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam▼ in Type 1 spinal muscular atrophy (SMA)​
Laurent Servais, Giovanni Baranello, Odile Boespflug-Tanguy, John W Day, Nicolas Deconinck, Andrea Klein, Riccardo Masson, Maria Mazurkiewicz-Beldzinska, Eugenio Mercuri, Kirsty Rose, Dmitry Vlodavets, Hui Xiong, Edmar Zanoteli, Muna El-Khairi, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Angela Dodman, Eleni Gaki, Basil T Darras, on behalf of the FIREFISH Working Group