A Look Ahead to 2019 – From Cure SMA President, Kenneth Hobby

By Cure SMA | Published On January 30, 2019

A Look Ahead to 2019 – From Cure SMA President, Kenneth Hobby

Dear SMA Community,

At the start of a new year, many of us begin to plan for the next 12 months. We anticipate the possibility of new achievements and look forward to new opportunities. In that spirit, we at Cure SMA wanted to share with you a look ahead to 2019.

We expect two major milestones in the SMA drug pipeline in 2019. In May, the FDA will issue a decision on Zolgensma, an IV gene therapy that we expect will be approved for infants with SMA type I. In the latter half of the year, Genentech/Roche plan to file for approval of risdiplam, an oral drug that increases the amount of SMN protein in the body.

This truly represents a new phase for those affected by SMA. Combined with the approval of Spinraza, individuals and families will have multiple therapies available to treat SMA. The future we have worked so long and hard to build is now taking shape.

We are committed in 2019 to doing everything we can to make sure that all individuals and families have access to these cutting-edge therapies, and to the care that they need. This means new progress on several fronts:

  • Continuing to advocate for broad and timely insurance coverage, so that all those affected by SMA have access to the treatment that best suits their goals and needs.
  • Increasing the number of sites that can provide these therapies, and removing administrative or other barriers that can delay delivery of treatment.
  • Educating families on the available therapies, so they can make the best treatment decision.

We also know that these changes mean local support and care are more important than ever. Individuals and families currently living with SMA must have access to customized support and programs in the communities where they live, attend work or school, and receive their treatment. This also means new progress on several fronts:

  • An expansion of our “Summit of Strength” program, a series of one-day local symposiums focused on the latest developments in treatment, support, and care. We will be holding 20 symposiums in 2019, with dates and locations to be announced shortly.
  • An expansion of our SMA Care Center Network, a clinical data registry that will help develop an evidence-based standard of care that will improve the lives of all those affected by spinal muscular atrophy. Already, seven sites have joined the care network, a number that will grow to two dozen by the end of 2019.
  • An expansion of our family support programs for all ages and stages of SMA, with a special focus on programs for our current community. This includes new support programs for adults living with SMA, a new and revised set of SMA care series booklets, and new resources to focus on the social impact of SMA.

We’ll also continue to expand our advocacy efforts. Already, 14 states have adopted newborn screening for SMA, with another three working on pilot projects. By the end of 2019, we expect that more than half of the states in the US will have adopted SMA newborn screening, and a number of those will be actively screening.

As we continue to expand our support programs, we also remain focused on the future of research. We continue to fund basic research to “seed” the SMA drug pipeline with new research into new targets and new ways of treating SMA. Our goal is that all those affected by SMA will have access to a combination of therapies that can be customized to their individual goals and needs.

This spring, we will announce $1 million in new grants. Spinraza, Zolgensma and risdiplam all work by increasing the amount of SMN protein in the body, and many researchers believe these therapies will be most effective when combined with therapies that address the muscles or other systems and pathways affected by SMA. In fact, three drugs that work on the muscles are currently being tested in clinical trials. We also continue our work to expand the number of sites participating in SMA clinical trials, so that these and other clinical trials can proceed as quickly and efficiently as possible.

Your continuous support and partnership has made these achievements possible. I look forward to keeping you updated on these and all our efforts throughout the year.

Very best,
Kenneth Hobby

Topics: Community & Awareness, About Us, Front Page News

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