News

May 7, 2018

AveXis Issues Community Statement on the SPR1NT Trial

AveXis Issues Community Statement on the SPR1NT Trial

AveXis has provided the following community statement on the SPR1NT trial.

Dear SMA Community,

AveXis, the gene therapy company developing a new approach to treat SMA known as AVXS‐101, recently treated the first patient in a new study known as SPR1NT. SPR1NT is a...

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Topics: Clinical Trials, Front Page News

May 2, 2018

Uniting the SMA Community Through Local Programs and Support

Uniting the SMA Community Through Local Programs and Support

After her son William's SMA type 2 diagnosis in April 2001, Heidi Johnson’s doctor immediately showed her the Cure SMA (then Families of SMA) website and said to look for support within the SMA community.

“After arriving home, we took the doc’s advice and opened up...

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Topics: Support & Care, About Us, Front Page News

May 1, 2018

Invitae Partners with Biogen to Offer Free Genetic Testing for SMA

Invitae Partners with Biogen to Offer Free Genetic Testing for SMA

Invitae Corporation recently announced that it will partner with Biogen to offer genetic testing at no charge to patients who may have...

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Topics: Support & Care, Research, Front Page News

April 30, 2018

Dr. Richard Finkel, M.D., Honored with the American Academy of Neurology's 2018 Sidney Carter Award in Child Neurology

Dr. Richard Finkel, M.D., Honored with the American Academy of Neurology's 2018 Sidney Carter Award in Child Neurology

Richard Finkel, M.D., the chief of neurology at Nemours Children’s Hospital in Orlando, Fla., was honored with the American Academy of Neurology’s 2018 Sidney Carter Award in Child Neurology, in recognition of his achievements in treating children with muscular dystrophy (MD) and...

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Topics: Research, Front Page News

April 25, 2018

AveXis to Present Phase 1 Data at the 70th American Academy of Neurology Annual Meeting

The Phase 1, open-label, dose-escalation trial was designed to evaluate the safety and tolerability of AVXS-101 in patients with SMA Type 1. The key measures of efficacy were the time from birth to an event and video...

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Topics: Clinical Trials, Front Page News

April 24, 2018

SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate

SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate

On Thursday, April 26, more than 70 chapter leaders, event organizers and board/committee members, representing 24 states and DC, will gather in Washington DC for two days of networking, learning, and advocacy. The activities include a day of advocacy on Capitol Hill, meeting with House and...

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Topics: Community & Awareness, Advocacy, Front Page News

April 24, 2018

The Discovery of Spinraza

The Discovery of Spinraza

SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein—called survival motor neuron protein or SMN protein—that is critical to the function of the nerves that control our...

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Topics: Our Impact, Research, Front Page News

April 23, 2018

There is Still Time to Complete Cure SMA's 2018 Community Survey

There is Still Time to Complete Cure SMA's 2018 Community Survey

Dear Members of the SMA Community,

For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate...

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Topics: Our Impact, Research, Front Page News

April 23, 2018

Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting

Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting

Biogen recently presented new data on Spinraza for the treatment of spinal muscular atrophy (SMA) during the 70th American Academy of Neurology (AAN) Annual Meeting, currently taking place from April 21-27 in Los Angeles, California. Both platform and poster presentations highlighted the...

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Topics: Clinical Trials, Research, Front Page News

April 18, 2018

Spring 2018 Issue of Compass Now Available Online

Spring 2018 Issue of Compass Now Available Online

The Spring 2018 issue of Compass is now available online. The issue covers Cure SMA’s newborn screening efforts, including how NBS can help children diagnosed with SMA, the addition of SMA to the federal Recommended...

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Topics: Research, Advocacy, Front Page News

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