Symptomatic

Diagnosed just before 3 months
and treated with onasemnogene abeparvovec-xioi shortly after.

Wesley

Pre-Symptomatic

Because of family history, he was diagnosed in utero and treated with onasemnogene abeparvovec-xioi weeks after birth.

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Kafele, Jr.

Wesley

Clinical

Family

Value

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Kafele

Gene Therapy - Clinical:

"Highlights of the clinical trial program include 91% survival without permanent breathing support at 14 months"

ZOLGENSMA® (onasemnogene abeparvovec-xioi): Suspension for Intravenous Infusion. Symptomatic clinical study results. Novartis Gene Therapies, Inc. 2021. www.zolgensma.com/clinical-studies/symptomatic-study-results.

Outlook - Clinical:

"Interim results of a long-term follow-up study show continued response to the gene therapy, with some patients even gaining the ability to stand up"

“100% maintained motor milestones”

ZOLGENSMA® (onasemnogene abeparvovec-xioi): Suspension for Intravenous Infusion. START Long-Term Follow-Up: ZOLGENSMA®(onasemnogene abeparvovec-xioi) demonstrates durability of effect over 5 years after treatment. Novartis Gene Therapies, Inc. 2021. www.zolgensma-hcp.com/clinical-experiences/start-long-term-follow-up-study-efficacy.

Value:

“Medical costs of roughly $1 billion per year”

Burns JK, Kothary R, Parks RJ. Opening the window: The case for carrier and perinatal screening for spinal muscular atrophy. Neuromuscul Disord. 2016;26(9):551-559. https://www.sciencedirect.com/science/article/pii/S0960896615301528

“Cost per patient, excluding disease-altering therapies, ranges from about $75,000 to about $200,000 per year”

Dangouloff T, Botty C, Beaudart C, et al. Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments. Orphanet J Rare Dis. 2021;16:47. https://doi.org/10.1186/s13023-021-01695-7.

Wesley

Outlook - Clinical:

"An ongoing long-term follow-up study will provide some answers, but it is clear that both Wesley and Sienna have exceeded developmental expectations for untreated children with SMA type 1"

Treatment - Clinical:

“In one ongoing study, many patients with SMA were able to reach developmental milestones like sitting up, standing for at least ten seconds, and even walking”

ZOLGENSMA® (onasemnogene abeparvovec-xioi): Suspension for Intravenous Infusion. Efficacy: SPR1NT: An open-label, single-arm clinical trial of presymptomatic patients with SMA. Novartis Gene Therapies, Inc. 2021. www.zolgensma-hcp.com/clinical-experiences/spr1nt-trial-efficacy.