News

October 18, 2018

AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy
Type I

AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy <br> Type I

AveXis, Inc., a Novartis company, today announced that they have filed for FDA approval of AVXS-101, a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which is missing or mutated in individuals with SMA.

This first filing is for intravenous (IV) delivery of gene...

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Topics: Clinical Trials, Front Page News

October 17, 2018

North Carolina to Screen Newborns for SMA Through Early Check

North Carolina to Screen Newborns for SMA Through Early Check

Early Check, a new research study led by RTI International, is now available for newborn babies in North Carolina. Early Check is a free screening study designed to identify children with rare health conditions before symptoms appear and study the benefits of early treatments. New and...

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Topics: Our Impact, Advocacy, Front Page News

October 17, 2018

Breakthrough Prize Awarded to Adrian Krainer and Frank Bennett for SMA Research Leading to Spinraza

Breakthrough Prize Awarded to Adrian Krainer and Frank Bennett for SMA Research Leading to Spinraza

Collaborators Adrian Krainer, of Cold Spring Harbor Laboratory, and C. Frank Bennett, from Ionis Pharmaceuticals, received the 2019

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Topics: Research, Front Page News

October 10, 2018

Cure SMA Receives Generous Funding from Luke 18:1 Foundation

Cure SMA Receives Generous Funding from Luke 18:1 Foundation

Cure SMA would like to thank the Luke 18:1 Foundation for their generous donations to our equipment pool and research programs. The foundation's generosity will provide support and hope to all those affected...

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Topics: Community & Awareness, Support & Care, Front Page News

October 9, 2018

SMA Care Center Network and Clinical Data Registry Launched

SMA Care Center Network and Clinical Data Registry Launched

Cure SMA today announced the launch of our SMA Care Center Network. The SMA Care Center Network is the centerpiece of our efforts to address the changing landscape of SMA. The goal of the SMA Care Center Network is to develop an...

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Topics: Community & Awareness, Support & Care, Front Page News

October 8, 2018

New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA

New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA

Biogen, Inc. announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA® (nusinersen) in 25...

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Topics: Clinical Trials, Research, Front Page News

October 5, 2018

Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA

Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA

Cytokinetics, Incorporated and Astellas Pharma Inc. today provided an update for their collaborative skeletal muscle program, including clinical trial updates for reldesemtiv in SMA.

In June, Cytokinetics announced data from a Phase...

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Topics: Clinical Trials, Research, Front Page News

October 3, 2018

Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress

Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH and...

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Topics: Clinical Trials, Research, Front Page News

October 3, 2018

Genentech Releases Community Statement on Risdiplam

Genentech Releases Community Statement on Risdiplam

The following statement is a community update from Genentech/Roche on the risdiplam studies FIREFISH and SUNFISH. 

Dear SMA community,

We are happy to share an update on our FIREFISH and SUNFISH studies of risdiplam. We would also like to acknowledge the tremendous support...

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Topics: Clinical Trials, Research, Front Page News

September 27, 2018

Community Spotlight: Adrienne Vollmer

Community Spotlight: Adrienne Vollmer

In May, Governor Eric Holcomb held a signing ceremony for Indiana House Bill 1017, adopting newborn screening for SMA and SCID. Dubbed “Graham’s Bill” in honor of Graham Vollmer, the bill was largely advanced by the efforts of...

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Topics: Community & Awareness, Advocacy, Front Page News

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