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The Critical Path Institute (C-Path) is a non-profit organization dedicated to the facilitation of drug development. This request is submitted by the Rare Diseases Cures Accelerator Data and Analytics platform (RDCA-DAP), C-Path’s rare diseases platform. RDCA-DAP is a neutral platform that houses patient data from diverse sources, including clinical trials, patient registries and hospital medical records across a multitude of rare diseases. RDCA-DAP’s objective is to promote the sharing of already collected patient data, and to help data contributors improve their future data collection. RDCA-DAP hosts such data in a format ready for regulatory submissions but also suitable for scientific and analytical work. By doing so, the RDCA-DAP aims to accelerate the understanding of disease progression, the development of better clinical and biological measures to assess patients’ diseases, and to facilitate the design of innovative clinical trials. RDCA-DAP is positioned to generate solutions that will facilitate the development of new drugs for rare diseases patients. As such, the utility of patient data is maximized, and the data may be leveraged to develop tools accessible by clinicians (from academia and industry) that will optimize and accelerate drug development across rare diseases. RDCA-DAP key objectives include: Development of a database of patient data across all rare diseases comprised of data from a wide range of sources that can be searched in multiple ways and used for analysis. The database is secured and protects patients’ identity from being revealed. Sharing of data with the global research community through a secure interface with appropriate privacy controls. Data may be used within the platform and/or some data may be available for download in formats suitable for submissions to regulatory authorities (Food and Drug Administration (FDA), European Medicines Agency (EMA)) Aiding in the analysis of the data by providing analytical functionalities and tool in the platform, as well as opportunities to collaborate with C-Path data science team. Connecting RDCA-DAP with other platforms hosting patient data, including Vivli, to ensure the platform does not work in isolation. In turn, this will increase the amount of data available to scientists and maximize opportunities for innovation and science. RDCA-DAP aims to be a diseases agnostic effort to help as many rare diseases as possible. In the spirit of expanding our database and helping the advancement of our solutions to facilitate drug development for neuromuscular diseases (NMD), we selected several Spinal Muscular Atrophy (SMA) datasets (NCT01494701, NCT01780246, NCT02052791, NCT01703988, NCT01839656, NCT02193074, NCT02292537 NCT02462759). SMA is a genetic disorder that causes muscle weakness and wasting due to the loss of motor neurons in the spinal cord, with an estimated worldwide prevalence of 1 in 10,000 births. Life- expectancy of SMA patients have increased in recent years with new therapies made available but remains a diseases with significant unmet needs. The data will be fully incorporated into RDCA-DAP for future analyses, and external sharing to RDCA-DAP users, as defined by Vivli, Biogen and RDCA-DAP. We selected several SMA datasets to expand the pilot of mutual sharing of rare diseases data between RDCA-DAP and Vivli. RDCA-DAP currently host data over 41 different rare diseases, but the sharing of these data will help us increase our neuromuscular diseases database. The data will reside in our aggregated database already enriched in rare dystrophies (Duchenne, Limb-Girdle Muscular Dystrophy, Facioscapulohumeral muscular Dystrophy, etc.) and neuromuscular diseases (Amyotrophic Lateral Sclerosis, Charcot-Marie-Tooth). This will provide critical new data that will be leveraged by our users to develop analytical work that will improve understanding of SMA, hopefully advancing more treatments for SMA and related NMD.