News

October 18, 2018

AveXis Issues Community Statement on FDA Filing for SMA Type I

AveXis has provided the following community statement on their FDA filing for SMA type I.

Dear SMA Community,

AveXis, a Novartis company, is pleased to let you know that we have submitted regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important and exciting initial...

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Topics: Clinical Trials, Front Page 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 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

September 26, 2018

Cure SMA Launches a Family Support App for Your Mobile Device!

Cure SMA Launches a Family Support App for Your Mobile Device!

Cure SMA is excited to announce the launch of a new mobile app for the SMA community. The Cure SMA Guide app is a family support program that takes a new approach to provide useful tools and information related to SMA care, to use at home and on-the-go. The app is now...

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

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