News

April 17, 2019

AveXis Data Reinforces Effectiveness of Zolgensma® in Treating SMA Type 1

AveXis Data Reinforces Effectiveness of Zolgensma® in Treating SMA Type 1

AveXis, a Novartis company, today announced that interim data from its Phase 3 STR1VE trial of Zolgensma®(AVXS-101) in spinal muscular atrophy Type 1 showed prolonged event-free survival,...

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Topics: Clinical Trials, Research, Front Page News

April 17, 2019

Biogen Presents New Data Regarding Adults and Infants with SMA

Biogen Presents New Data Regarding Adults and Infants with SMA

Biogen has announced it will present new data illustrating the rapidly progressive nature of spinal muscular atrophy (SMA) in adults, adolescents and older...

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Topics: Clinical Trials, Research, Front Page News

March 26, 2019

The Winter 2019 issue of Compass in Now Available Online

The Winter 2019 issue of Compass in Now Available Online

The Winter 2019 issue of Compass is now available online. This issue covers Cure SMA’s latest drug discovery grant funding and features an updated SMA drug pipeline.

Drug discovery, also called translational...

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Topics: Clinical Trials, Research, Front Page News

March 5, 2019

Biogen Provides 2019 Community Update on Spinraza

Biogen Provides 2019 Community Update on Spinraza

Dear members of the SMA community,

As we celebrate Rare Disease Day 2019, we are honored to continue supporting the spinal muscular atrophy (SMA) community. In recent weeks and months, we engaged the community on a number of different initiatives and are excited to share updates on our...

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Topics: Clinical Trials, Research, Front Page News

March 1, 2019

Genentech/Roche and Cure SMA to Co-Host Webinar

Genentech/Roche and Cure SMA to Co-Host Webinar

On Monday, March 11th, at 12pm CST (10am PST/11am MST/1pm EST) Genentech/Roche and Cure SMA will co-host a webinar for the SMA community. During the webinar, you’ll hear from Genentech/Roche on their history working with the SMA community, and their investigational medicine risdiplam....

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Topics: Clinical Trials, Research, Front Page News

February 26, 2019

Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans

Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans

Scholar Rock today announced positive interim results from the ongoing Phase 1 single- and multiple-ascending dose clinical trial of SRK-015 in...

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Topics: Clinical Trials, Research, Front Page News

January 22, 2019

Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA

Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA

Cytokinetics, Incorporated (CYTK) today announced that it has received feedback from the U.S. Food and Drug Administration (FDA) that the Six Minute Walk...

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Topics: Clinical Trials, Research, Front Page News

January 7, 2019

Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam

Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam

On January 7th, PTC Therapeutics, Inc. provided a corporate update, which will be detailed as part of the company's presentation at the 37th Annual J.P. Morgan...

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Topics: Clinical Trials, Research, Front Page News

December 26, 2018

December Webinar on SMA Treatments and Clinical Trials Now Available Online

December Webinar on SMA Treatments and Clinical Trials Now Available Online

A recording of the December 13th webinar updating the community on SMA treatments and clinical trials is now available online. A PDF of the webinar presentation is also available for...

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Topics: Clinical Trials, Research, Front Page News

December 13, 2018

Biogen Issues Q4 Community Statement on Spinraza

Biogen Issues Q4 Community Statement on Spinraza

Bigoen has provided the below community statement on Spinraza. 

Dear Members of the SMA community,

As we approach the two-year anniversary of the U.S. Food and Drug Administration’s (FDA) approval of SPINRAZA® (nusinersen), we want to thank you for your ongoing...

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Topics: Clinical Trials, Research, Front Page News

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