Author name: Zach Galati

Cure SMA and the Luke 18:1 Foundation Launch New Fold & Go Wheelchair Grant Program

Cure SMA is excited to announce the launch of a brand-new equipment grant program, made possible through the generous support of the Luke 18:1 Foundation. This program will provide Fold & Go Travel Electric Wheelchairs to children and adults living with SMA that they will own outright, not borrow. Unlike our current equipment pool loan […]

Cure SMA and the Luke 18:1 Foundation Launch New Fold & Go Wheelchair Grant Program Read More »

Cure SMA Update on SMA Treatment Delays

Dear SMA Community, Yesterday was a disappointing and frustrating day for our community. We learned of two FDA approval delays for Scholar Rock’s apitegromab and Biogen’s high-dose nusinersen/Spinraza. While there are many unknowns regarding timelines and next steps, the most encouraging news in all of this is that the issues named by the FDA are

Cure SMA Update on SMA Treatment Delays Read More »

Biogen Receives News on Supplemental New Drug Application (sNDA) for High Dose Nusinersen from the U.S. FDA

Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the supplemental New Drug Application (sNDA) for the high-dose regimen of nusinersen (SPINRAZA) for people living with spinal muscular atrophy (SMA). The CRL requests updates to the technical information included in the Chemistry Manufacturing

Biogen Receives News on Supplemental New Drug Application (sNDA) for High Dose Nusinersen from the U.S. FDA Read More »

Scholar Rock Receives News on Biologics License Application (BLA) for Apitegromab from the U.S. FDA

Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA) for the treatment of people living with spinal muscular atrophy (SMA). The CRL is related to observations identified during a routine inspection of Catalent Indiana LLC,

Scholar Rock Receives News on Biologics License Application (BLA) for Apitegromab from the U.S. FDA Read More »

Cure SMA 2025 Hill Day: 24 Hours of Advocacy Impact

On Tuesday, September 16, about 150 adults with spinal muscular atrophy (SMA) and family members of children with SMA visited Capitol Hill to advocate for key priorities of the SMA community. Advocates from 34 states met in-person with 155 congressional offices in Washington, DC, including 87 U.S. House offices and 68 U.S. Senate offices, to

Cure SMA 2025 Hill Day: 24 Hours of Advocacy Impact Read More »

2025 Hope on the Hill Celebrates Day of Advocacy in Washington, DC

Cure SMA celebrated a day of advocacy on Capitol Hill on September 16 with more than 170 individuals with spinal muscular atrophy (SMA), their families, and SMA community partners at the 2025 Hope on the Hill reception. Founded in 2011, Hope on the Hill was created to raise awareness of SMA in Washington, DC, and

2025 Hope on the Hill Celebrates Day of Advocacy in Washington, DC Read More »

SMA Researchers and Clinicians from Around the World Gather at the 29th Annual SMA Research & Clinical Care Meeting (2 of 2)

At the end of June, SMA researchers and clinicians from around the world gathered in Anaheim, California, for the 29th Annual SMA Research & Clinical Care Meeting. Their common goals were to accelerate the pace of research into SMA treatments and to achieve excellence in SMA clinical care. The annual meeting provides an opportunity for

SMA Researchers and Clinicians from Around the World Gather at the 29th Annual SMA Research & Clinical Care Meeting (2 of 2) Read More »

SMA Researchers and Clinicians from Around the World Gather at the 29th Annual SMA Research & Clinical Care Meeting (1 of 2)

At the end of June, SMA researchers and clinicians from around the world gathered in Anaheim, California, for the 29th Annual SMA Research & Clinical Care Meeting. Their common goals were to accelerate the pace of research into SMA treatments and to achieve excellence in SMA clinical care. The annual meeting provides an opportunity for

SMA Researchers and Clinicians from Around the World Gather at the 29th Annual SMA Research & Clinical Care Meeting (1 of 2) Read More »

Cure SMA Shares Patient Voice and Needs with FDA

  One of Cure SMA’s top priorities is to relay the SMA community’s experiences, preferences, and unmet needs to the United States Food and Drug Administration (FDA). This enables the FDA to make patient-centered decisions about new SMA drugs. This is a critical time for patient input on SMA treatment, as new therapies and variations

Cure SMA Shares Patient Voice and Needs with FDA Read More »

Cure SMA Educates Insurers on Unmet Needs and Access Barriers of the SMA Community

  Cure SMA completed a year-long advocacy effort to educate public and private insurers about the current state of spinal muscular atrophy (SMA), including the unmet needs of individuals with SMA and barriers they face in accessing essential healthcare and treatments. Patient and clinical-reported data have chronicled how SMA is rapidly changing due to the

Cure SMA Educates Insurers on Unmet Needs and Access Barriers of the SMA Community Read More »

Scroll to Top