New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA

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Scholar Rock yesterday announced new quality-of-life (QoL) data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment which indicate stabilization or continued improvement with apitegromab for nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) receiving an SMN-targeted therapy. The TOPAZ trial assessed activities of daily […]

New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA Read More »

Community Spotlight: Amber-Joi Watkins and Family

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The Igbo and Yoruba proverb, “It takes a village to raise a child” has been in use for centuries and remains in use across languages and cultures due to its continued relevance. For Amber-Joi Watkins, her husband, Tommy Domalski, and their daughter, Céline, that village includes family and friends, as well as Céline’s pediatrician, her

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Cure SMA Holds Patient-Led Listening Session with FDA

Leave a Comment / Community Awareness, Latest News, Our Impact, Research /

On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their unmet medical needs. This Patient-Led Listening Session was a private teleconference attended by patient advocates, FDA representatives from the Center for Drug

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A Deliciously Easy Way to Fundraise for Cure SMA

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Nothing brings people together more than sharing a meal, but what if you could make that meal even more meaningful? We’re encouraging all members of our SMA community to consider hosting a dining fundraiser, where a portion of purchases are designated to support Cure SMA. This is also a great way to supplement your efforts

A Deliciously Easy Way to Fundraise for Cure SMA Read More »

Cure SMA Holds Patient-Led Listening Session with FDA

Leave a Comment / Community Awareness, Our Impact, Research /

On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their unmet medical needs. This Patient-Led Listening Session was a private teleconference attended by patient advocates, FDA representatives from the Center for Drug

Cure SMA Holds Patient-Led Listening Session with FDA Read More »

A Deliciously Easy Way to Fundraise for Cure SMA

Leave a Comment / Events & Fundraising, Front Page News /

Nothing brings people together more than sharing a meal, but what if you could make that meal even more meaningful? We’re encouraging all members of our SMA community to consider hosting a dining fundraiser, where a portion of purchases are designated to support Cure SMA. This is also a great way to supplement your efforts

A Deliciously Easy Way to Fundraise for Cure SMA Read More »

Special NBS Awareness Month Announcement: South Carolina Screens for SMA!

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Cure SMA is pleased to announce that South Carolina is now screening babies born in the state for spinal muscular atrophy (SMA). South Carolina Department of Health and Environmental Control (DHEC) officials shared the exciting news with Cure SMA in a written statement earlier today: Good news! The new multiplex PCR based method for screening

Special NBS Awareness Month Announcement: South Carolina Screens for SMA! Read More »

Special NBS Awareness Month Announcement: South Carolina Screens for SMA!

Leave a Comment / Advocacy, Our Impact /

Cure SMA is pleased to announce that South Carolina is now screening babies born in the state for spinal muscular atrophy (SMA). South Carolina Department of Health and Environmental Control (DHEC) officials shared the exciting news with Cure SMA in a written statement earlier today: Good news! The new multiplex PCR based method for screening

Special NBS Awareness Month Announcement: South Carolina Screens for SMA! Read More »

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