Cure SMA

On Friday, December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.

Join them as they surf to wipe out spinal muscular atrophy (SMA).

Over the past few months, Cure SMA has announced a total of just over $2 million in new research funding.

Cure SMA has awarded a $304,000 drug discovery grant to Livio Pellizzoni, PhD, at Columbia University. The award is for the project, "Pharmacological Inhibition of p38αMAPK as a Candidate Therapeutic Approach for SMA." About the Project Dr.

Cure SMA has awarded a $300,000 drug discovery grant to Charlotte Sumner, MD, at Johns Hopkins University, in collaboration with Imago Pharmaceuticals.

Cure SMA has awarded a $70,000 research grant to Dr. Christine DiDonato at Lurie Children's Hospital of Chicago for her project, "Assessing mediators of muscle weakness in SMA mice." Individuals with SMA do not produce survival motor neuron (SMN) protein.

Cure SMA has awarded a $140,000 research grant to Charlotte Sumner, MD, at Johns Hopkins University for her project, "Assessing the reversibility of proximal axon abnormalities in SMA mice." Axons are long projections that grow out of motor neurons and...

Cure SMA has awarded a $50,000 clinical care research grant to Melissa Alderfer, PhD, at the Alfred I.

Cure SMA has awarded a $50,000 clinical care research grant to Tariq Rahman, PhD, at the Alfred I.

Cure SMA has awarded a $30,000 research grant to Jocelyn Cote, PhD, at the University of Ottawa for his project, "The role of SMN as a translational regulator." The grant to Dr. Cote is funded by Families of SMA Canada.