Cure SMA Announces the Launch of Phase 2 SMA Industry Collaboration

By: PRLog
ELK GROVE VILLAGE, Ill. - June 20, 2017 - PRLog -- Cure SMA, a leading national non-profit organization dedicated to the treatment and cure of spinal muscular atrophy, today announced the launch of Phase 2 of the Spinal Muscular Atrophy (SMA) Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that are critical to advancing drug development in SMA and will benefit the broader SMA community.

Spinal muscular atrophy is a neuromuscular disease caused by a mutation in the gene survival motor neuron 1 (SMN1). In a healthy person, this gene produces a protein that is critical to the function of the nerves that control muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness. SMA is currently the leading genetic cause of death for children under age two.

The six pharmaceutical companies that make up the SMA Industry Collaboration — Astellas, AveXis, Biogen, Genentech/Roche, Cytokinetics, and Ionis — are currently engaged in drug development for SMA.

In the first phase of the collaboration, the group coalesced around three major topics of importance to the SMA community: engagement with the FDA and other regulatory agencies, education in the medical community to reduce time to diagnosis, and increasing clinical trial site capacity.

The signature accomplishment of this first phase was the execution of a successful Patient Focused Drug Development Meeting with the FDA, a project of the regulatory topic group.

In this second phase, the regulatory group will oversee the development of the Voice of the Patient Report as part of the follow-up to the PFDD meeting. The group will also work on plans for future engagement with the FDA.

In addition, the emphasis in this second phase will shift toward the other two topic groups, which have spent the first phase laying the foundation for their activities.

The education topic group will work on expanding awareness of SMA among families and caregivers as well as pediatricians and other healthcare providers, with a focus on reducing diagnostic delays. Diagnostic delays can put significant financial, logistical and emotional strain on families. More importantly, they can also reduce the opportunity for early treatment. The education topic group will work with stakeholders to improve recognition of SMA, to shorten the time to diagnosis and avoid unnecessary delays in beginning treatment.

The clinical trials group will focus on identifying and pursuing opportunities to increase site capacity and optimize readiness throughout the US. The goal is to improve the SMA community's access to clinical trial sites and increase capacity. In this phase, the topic group will focus both on assessing and improving existing clinical trial networks, as well as reaching out to potential new SMA clinical trial sites.

The activities of the SMA Industry Collaboration are jointly supported by Astellas, AveXis, Biogen, Genentech/Roche, Cytokinetics, and Ionis.

To learn more, visit www.curesma.org.

Contact
Sarah McCall
***@curesma.org

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