December 2017

Biogen and Ionis Enter Into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy

Biogen and Ionis Pharmaceuticals announced that they have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA). Biogen will have the option to license therapies arising out of this collaboration and will be responsible for their development and commercialization. “We are pleased to […]

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Cure SMA Assists Hundreds of Families in 2017

Each year, Cure SMA provides thousands of families with vital support and resources to help them live active, engaged and hopeful lives. Our programs provide tangible support for families affected by SMA, easing the difficulties of an SMA diagnosis, and showing each family that they are not alone and that there is hope. Our family

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Miller McNeil Woodruff Foundation Awards Cure SMA Generous Gift

The Miller McNeil Woodruff Foundation recently awarded $87,000 to Cure SMA, in order to further research that will lead to treatments for all ages, stages and types of SMA. Their gift will also help fund continued advocacy and awareness initiatives. The generous donation is made each year by Patrick and Meredith Woodruff, in honor of

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Cure SMA Statement on Tax Reform Bill and the Repeal of the Individual Mandate

On Wednesday, Congress passed the most sweeping tax bill in a generation. Cure SMA has been monitoring and advocating on your behalf as this legislation went through the Congress. We are happy to share that: Tax credits for rare disease drugs are saved. Cure SMA partnered with other rare disease groups to save a tax

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Fall 2017 Directions Now Available for Download

The Fall 2017 issue of Directions is now available. Please visit our Support & Care Publications page to download this issue or past issues of Directions. Our biannual newsletter builds our community by sharing stories and experiences. Families from all over the world connect with each other through Cure SMA to organize, share stories, photos,

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Cure SMA Advocates Continue to Advance Newborn Screening Efforts

SMA advocates across the country are working tirelessly with Cure SMA to educate state decision makers on newborn screening. In Maryland and Minnesota, the states’ Newborn Screening Advisory Committees voted to approve a recommendation to add SMA to the state panel. We now await final approval by both states’ Commissioners of Health. Illinois, Ohio and

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AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that, based on review of data submitted, the company may initiate its planned Phase 1 clinical trial of AVXS-101 for patients with spinal

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Adult Scholarships Offered for the 2018 Annual SMA Conference

Cure SMA is pleased to offer scholarships to the 2018 Annual SMA Conference for all adults with SMA, thanks to a generous grant from The Dhont Family Foundation. The scholarship covers registration for one adult, age 18+, and one aide. Each year, we look forward to reuniting as a community at the Annual SMA Conference.

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Cure SMA-Funded Research Results in 21 Published Journal Articles

An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who receive Cure SMA funding often publish their findings in peer-reviewed journals. Published articles allow the experiments and results to be reviewed and vetted by other scientists, who may then use

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