Author name: Zach Galati

Biogen Receives FDA Approval of High Dose SPINRAZA for the Treatment of SMA

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Biogen today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for the High Dose regimen of SPINRAZA (nusinersen) to treat people living with spinal muscular atrophy (SMA). With broad label approval for all those living with SMA, this is an excellent result for the SMA community.  SPINRAZA (nusinersen) is an antisense oligonucleotide […]

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Cure SMA Awards $150,000 Grant to Tetsuya Akiyama, MD, PhD, Stanford University School of Medicine

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Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal muscular atrophy (SMA) biology. Our Scientific Advisory Board ranks the submitted proposals based on their scientific merit and their alignment with Cure SMA’s research priorities. Funding is then awarded to

Cure SMA Awards $150,000 Grant to Tetsuya Akiyama, MD, PhD, Stanford University School of Medicine Read More »

Cure SMA Expands Travel Support Package Eligibility to Include Teens with SMA

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Cure SMA is excited to share an important update to our travel support programs. In response to community feedback and the growing need for accessible travel support for younger individuals, we have expanded eligibility for our Travel Support Package to now include teens with SMA. Previously available only to adults with SMA, the Travel Support

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Genentech Issues Community Letter Regarding Emugrobart

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Today, Genentech announced the discontinuation of emugrobart (an investigational anti-myostatin antibody, also known as GYM329) into Phase III development for spinal muscular atrophy (SMA). The decision to stop clinical development of emugrobart in SMA follows a comprehensive assessment of the totality of the evidence from Part 1 of the MANATEE ( NCT05115110 ) study –

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Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh in Scotland

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Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal muscular atrophy (SMA) biology. Our Scientific Advisory Board ranks the submitted proposals based on their scientific merit and their alignment with Cure SMA’s research priorities. Funding is then awarded to

Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh in Scotland Read More »

Evrysdi Risdiplam Tablet Is Now Approved For Broader Use In SMA

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We’re excited to share an important update for the spinal muscular atrophy (SMA) community. The U.S. Food and Drug Administration (FDA) has approved a label expansion for Evrysdi® (risdiplam) tablets, effective February 10, 2026. The tablet formulation is now approved for administration via nasogastric (NG) or gastrostomy (G) feeding tubes after dispersion in water. This

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Cure SMA Awards $150,000 Grant to Christian Simon, PhD, at Leipzig University in Germany

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Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal muscular atrophy (SMA) biology. Our Scientific Advisory Board ranks the submitted proposals based on their scientific merit and their alignment with Cure SMA’s research priorities. Funding is then awarded to

Cure SMA Awards $150,000 Grant to Christian Simon, PhD, at Leipzig University in Germany Read More »

Novartis Releases Winter SMA Community Update Letter

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Novartis recently shared a Winter 2026 update highlighting the FDA approval of ITVISMA® (onasemnogene abeparvovec-brve) for intrathecal use in people ages 2 and older with SMA, offering a one-time gene replacement therapy option. The update also includes ongoing engagement opportunities with the SMA community, upcoming ITVISMA webinars, Adaptive Sports and Recreation resources from Cure SMA,

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Cure SMA Awards $75,000 Grant to Jiangbing Zhou, PhD, at Yale University

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Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal muscular atrophy (SMA) biology. Our Scientific Advisory Board ranks the submitted proposals based on their scientific merit and their alignment with Cure SMA’s research priorities. Funding is then awarded to

Cure SMA Awards $75,000 Grant to Jiangbing Zhou, PhD, at Yale University Read More »

Cure SMA Awards $150,000 Grant to Emma Sutton, PhD, at Ottawa Hospital Research Institute in Canada

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Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal muscular atrophy (SMA) biology. Our Scientific Advisory Board ranks the submitted proposals based on their scientific merit and their alignment with Cure SMA’s research priorities. Funding is then awarded to

Cure SMA Awards $150,000 Grant to Emma Sutton, PhD, at Ottawa Hospital Research Institute in Canada Read More »

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