News

July 10, 2018

Roche Releases Community Update on Risdiplam

Roche Releases Community Update on Risdiplam

The following is a community update from Genentech/Roche on the risdiplam (RG7916) program.

Dear SMA advocacy community,

As requested, we are very pleased to share an update on the RG7916 program.

We would also like to extend a special thanks to study participants and...

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Topics: Clinical Trials, Research, Front Page News

June 19, 2018

PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings

On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy...

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Topics: Clinical Trials, Research, Front Page News

June 16, 2018

Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference

Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference

Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by...

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Topics: Clinical Trials, Research, Front Page News

June 1, 2018

Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing...

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Topics: Clinical Trials, Research, Front Page News

May 30, 2018

Roche Releases Community Statement on Olesoxime Program

Roche Releases Community Statement on Olesoxime Program

Roche today provided the following community statement on olesoxime.

Dear members of the SMA Community,

We would like to provide you with an update about olesoxime, an investigational molecule in SMA.

Since we bought olesoxime from Trophos in 2015, we have had many...

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Topics: Clinical Trials, Research, Front Page News

May 22, 2018

Biogen Releases Community Statement on Spinraza Access and New Data

Biogen Releases Community Statement on Spinraza Access and New Data

Biogen has released the following community statement on Spinraza access and new research data.

Dear Members of the SMA community,

Thank you for your continued support, efforts to raise awareness about SMA, and participation in clinical trials and studies as we seek to expand...

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Topics: Clinical Trials, Research, Front Page 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

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 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 16, 2018

Genentech to Present New RG7916 Data at the American Academy of Neurology (AAN) Annual Meeting

Genentech to Present New RG7916 Data at the American Academy of Neurology (AAN) Annual Meeting

Genentech/Roche announced today that new data on its approved and investigational medicines for neurological conditions will be presented during the 70th American Academy of Neurology (AAN) Annual Meeting from April 21-27 in Los Angeles, California. The data will include investigational...

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Topics: Clinical Trials, Research, Front Page News

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