Community Statement: Quarterly Update from Novartis Gene Therapies

Dear Members of the SMA Community,

Novartis Gene Therapies is committed to working with our partners in the SMA community to make a difference in the diagnosis, treatment, and care of those with SMA.  Through our engagement with you directly, and through our collaborations with Cure SMA, together we can make a difference. We are proud of the work we are doing together to ultimately improve patient outcomes.

During the first quarter of 2021, Novartis Gene Therapies supported a number of Cure SMA collaboration projects. The projects, focused on areas of common cause, include education of caregivers and healthcare providers, gathering data about how treatments are being used in the real world (versus in clinical trials), and improving the way we inform people about and conduct our clinical trials. Much of this important work is captured in the latest Cure SMA Directions Community Newsletter. It’s also been our privilege to connect with the community in training and spreading awareness at the state and local level, and at Cure SMA Chapter and Leadership meetings.

At Novartis Gene Therapies, we remain focused on our role to support caregivers and equip them with pre-natal and early diagnosis education. We believe it is vital that we broaden the awareness of SMA and the urgency to treat by sharing the positive impact receiving early treatment has had on families of SMA. As a sponsor of this year’s Cure SMA Annual SMA Conference, we will continue this advocacy and education through virtual healthcare provider and caregiver events, social media, and a caregiver-led roundtable discussion.

We continue to pursue solutions for patients with all types of SMA and as such continue to invest in clinical trials and new research. Thank you for your partnership.


Novartis Gene Therapies

US-ZOL-21-0137 04/2021


Last month, Novartis Gene Therapies announced it will initiate SMART, a Phase 3b clinical study to further evaluate the safety and efficacy of Zolgensma in patients with SMA. Clinical data from this study will supplement emerging real-world evidence and use of Zolgensma. Learn more here.

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