Mary Schroth, MD: The Impact of FDA Approvals on SMA Advocacy


The chief medical officer at Cure SMA detailed the importance of having various treatment options for patients, particularly for adults with SMA.

“We’re just so excited that we’re making this a different disease now. SMA as I knew it as a clinician is going away, and I’m happy to say that.”

When a treatment is approved and subsequently introduced to the clinical landscape of disease, regardless of whether it is the first or the tenth agent for that disease state, at the very least, it offers patients something incredibly important: an option. In the case of a disease such as spinal muscular atrophy (SMA), where the number of therapies is rather limited, this impact is particularly monumental.

Each approval in SMA also offers those in positions of advocacy, such as Mary Schroth, MD, chief medical officer, Cure SMA, a unique opportunity. Schroth, a former pulmonologist, told NeurologyLive that the belief on their end is that no treatment is one-size-fits-all, so the more options, the better. Now, with the recent approval of risdiplam (Evrysdi; Genentech), patients with SMA will have an oral therapy available, joining the 2 other agents, AveXis’s gene therapy Zolgensma and Biogen’s nusinersen (Spinraza).

In this interview, Schroth detailed the importance of having options for patients and how certain administration routes and schedules—as well as differing indications—offer more personalized approaches, particularly for adult patients with SMA. She also explained Cure SMA’s role in advocating for patients to get treatment, and how each new agent impacts its ability to do so.

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