A bit more than half of the total funding will go directly to support local care. The remainder will fund basic research, newborn screenings, and clinical and regulatory initiatives. Part of the funding also is aimed at increasing patient access to treatments.
Specifically, this is how the $5 million investment will be used:
- Clinical Care Centers ($2.2 million) – Cure SMA is launching a network of Cure SMA Care Centers to improve patients’ care and quality of life. These centers are to jointly create a Cure SMA clinical data registry, which will be used to assist patients about health care. The network is expected to begin with 10 centers and gradually expand.
- Continued Funding for Clinical and Regulatory Research ($1 million) – Funding will be directed toward several key initiatives, such as engagement with the U.S. Food and Drug Administration (FDA) and other health regulatory agencies, increased education and awareness to the community, and expanding capacity for new clinical trial sites.
- Continued Funding for Basic Research ($900,000) – Investigating the causes and biology of SMA often proves more effective in finding new SMA drugs. Consequently, this funding aims to help address questions about survival motor neuron (SMN) protein, as well as other systems, pathways and processes that are affected in SMA.
- Improving Access to Treatments and Care ($400,000) – In order to increase capacity to follow, treat and evaluate SMA patients, the organization will award $50,000 grants to nine administration sites to help reduce barriers (including pre-authorization and insurance approvals, scheduling, and clinic support) that delay treatment and care access.
- SMA Newborn Screening Initiatives ($400,000) – With Spinraza (Nusinersen) now approved and available, and data suggesting that pre-symptomatic treatment improves clinical outcomes, it is vital to screen newborns as quickly as possible. This program will help fund resources needed for state public health labs in order to implement newborn screening for SMA.
- Training for Healthcare Providers to Improve Quality Care for SMA ($100,000) – Cure SMA will fund a new Therapist Mentoring Program to improve care in the local community, to provide information, experiences and resources for physical therapists and occupational therapists who are now entering the field of SMA, as well as to increase access to therapists and pulmonologists with expertise in SMA.
- Data Projects to Support Increased Access and Approvals ($100,000) – Earlier this year, a survey for families and patients was released to gather how SMA has impacted their lives. This portion of the investment will help release a similar survey in the coming months to track any changes in the burden of SMA over the past year, now that the first SMA treatment has been approved and is available.
Find out more about Spinraza and U.S. FDA approval here.