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

April 3, 2019

Cure SMA Awards $150,000 Grant to Jocelyn Cote, PhD, University of Ottawa

Cure SMA Awards $150,000 Grant to Jocelyn Cote, PhD, University of Ottawa

Cure SMA and Cure SMA Canada have awarded a $150,000 research grant to Jocelyn Cote, PhD, at the University of Ottawa for his project, “Investigating the contributions of CARM1 and HuR misregulation to SMA skeletal muscle and NMJ defects.”

While an essential role for SMN in...

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Topics: Research, Front Page News

March 28, 2019

Cure SMA Awards $200,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires

Cure SMA Awards $200,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires

Cure SMA and FAME (Families of SMA, Argentina) have awarded a $200,000 research grant to Alberto Kornblihtt, PhD, at the Universidad de Buenos Aires, Argentina, for his project, "Epigenetics in SMN2 E7 Alternative Splicing II.”

Epigenetics refers to changes that affect how much...

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Topics: Research, Front Page News

March 27, 2019

Cure SMA Awards $200,000 Grant to Charlotte Sumner, MD, Johns Hopkins University

Cure SMA Awards $200,000 Grant to Charlotte Sumner, MD, Johns Hopkins University

Cure SMA has awarded a $200,000 research grant to Charlotte Sumner, MD, at Johns Hopkins University, for her project, "Neurofilaments as markers of neurodegeneration in SMA.”

Dr. Sumner and her team are looking at the neuronal-specific cytoskeletal protein neurofilaments (NFs)...

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Topics: Our Impact, 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 21, 2019

Record Number of Abstracts Submitted for the 2019 SMA Researcher Meeting

Abstract submission is now closed for the 2019 SMA Researcher Meeting. We are excited to announce that we received 166 submissions, a record number. Our submissions come from researchers in 20 countries, 11 companies, and close to 65...

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Topics: Research, Front Page News

March 14, 2019

Cure SMA Launches Third Annual Community Survey to Address Important Issues in SMA Treatment

Cure SMA Launches Third Annual Community Survey to Address Important Issues in SMA Treatment

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 our priorities to the FDA...

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Topics: Community & Awareness, Research, Front Page News

March 8, 2019

Cure SMA Advocates for SMA Community at ICER Hearing

On Thursday, March 7, Cure SMA testified on behalf of the SMA community at a meeting of the Institute for Clinical and Economic Review (ICER). ICER is an independent health policy group that evaluates the effectiveness of drug treatments, and the costs associated with those treatments.

The purpose of the meeting was to review both...

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Topics: Research, Advocacy, 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

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