Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam

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On January 7th, PTC Therapeutics, Inc. provided a corporate update, which will be detailed as part of the company’s presentation at the 37th Annual J.P. Morgan Healthcare Conference. As part of the update, progress on regulatory filing for risdiplam was outlined. The risdiplam program is a collaboration between PTC, Genentech/Roche and SMA Foundation. The following […]

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Teen and Young Adult Survey Extended Through January 21st

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Are you or a family member between the ages of 12 and 25? Do you want to share your opinion on living with SMA and what is important to you regarding treatment options? In late November, Cure SMA launched the SMA Teen and Young Adult Clinical Meaningfulness Survey. Anyone with SMA between the ages of

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Community Spotlight: The Jankowski Family

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It has been a little over two years since doctors told Chris and Kelly Jankowski that their first-born son, William, had spinal muscular atrophy (SMA) type I. In the immediate wake of diagnosis, Cure SMA was able to help provide the Jankowskis with the resources to help them make decisions about care: asking the best

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Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration

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Cure SMA is pleased to announce the launch of an expanded Phase 3 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory

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Hope on the Hill Congressional Dinner Raises $200,000

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The 8th Annual “Hope on the Hill” Congressional Dinner was held on Tuesday, November 27, 2018 at The Willard Hotel in Washington, DC. More than 170 people from the SMA community – including government and industry partners – attended the dinner which raised $200,000 to fund advocacy efforts, research, and family support services. Since 2011,

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December Webinar on SMA Treatments and Clinical Trials Now Available Online

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A recording of the December 13th webinar updating the community on SMA treatments and clinical trials is now available online. A PDF of the webinar presentation is also available for download. An update on the status of dosing for Spinraza across the United States included the following information: As of October 2018, ~2,700 patients have

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Cure SMA Funding Leads to 16 New Research Publications in 2018

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An important goal of our research funding strategy is to share scientific findings with the broader scientific community. Scientists who receive Cure SMA funding often publish their findings in peer-reviewed journals. This means that other scientists can learn from their results, which will pay dividends across the wider landscape of SMA research—allowing us to multiply

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Community Spotlight: The Berkovits Family

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When Milo Berkovits was seven months old, his daycare teacher noticed he was not reaching his milestones. What followed from there on were visits to three different pediatricians, an early intervention assessment, a review by a private occupational therapist, another assessment by a private physical therapist and finally a neurologist visit. At nine months old,

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