Researchers Discover ‘Super Minigene’ that Helps Find Potential Therapies for Spinal Muscular Atrophy
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Researchers Discover ‘Super Minigene’ that Helps Find Potential Therapies for Spinal Muscular Atrophy

For decades researchers have had to isolate segments of genes in order to study them. They called the isolated gene a “mini gene.” Ravindra Singh, a biomedical science professor at…

Continue Reading Researchers Discover ‘Super Minigene’ that Helps Find Potential Therapies for Spinal Muscular Atrophy
Rare Community Profiles: Fiercely Advocating for the SMA Community: Why Amanda Will Never Stop Fighting for her Son Kayden
Photo courtesy of Amanda Chaffin

Rare Community Profiles: Fiercely Advocating for the SMA Community: Why Amanda Will Never Stop Fighting for her Son Kayden

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

Continue Reading Rare Community Profiles: Fiercely Advocating for the SMA Community: Why Amanda Will Never Stop Fighting for her Son Kayden
Rare Community Profiles: Singer-Songwriter Pidgie Uses Her SMA2 Experience to Advocate: “Music is Inclusion!”
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Rare Community Profiles: Singer-Songwriter Pidgie Uses Her SMA2 Experience to Advocate: “Music is Inclusion!”

Rare Community Profiles     Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…

Continue Reading Rare Community Profiles: Singer-Songwriter Pidgie Uses Her SMA2 Experience to Advocate: “Music is Inclusion!”
Treatment for Pre-symptomatic Spinal Muscular Atrophy Patients Granted FDA Priority Review
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Treatment for Pre-symptomatic Spinal Muscular Atrophy Patients Granted FDA Priority Review

PTC Therapeutics Inc. has just announced that their supplemental new drug application for Evrysdi (risdiplam) has been given priority review by the FDA. The aim is to expand the indication…

Continue Reading Treatment for Pre-symptomatic Spinal Muscular Atrophy Patients Granted FDA Priority Review