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!”
Singapore: 29,000 Donors Responded to a Plea By Devdan’s Parents For a One Time Gene Therapy Treatment That Costs $2.8 Million
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Singapore: 29,000 Donors Responded to a Plea By Devdan’s Parents For a One Time Gene Therapy Treatment That Costs $2.8 Million

According to YAHOO News, twenty-two-month-old Devdan has a rare disease called type 2 spinal muscular atrophy (SMA2) which affects nerve cells that control muscles. If left untreated progressive muscle weakness…

Continue Reading Singapore: 29,000 Donors Responded to a Plea By Devdan’s Parents For a One Time Gene Therapy Treatment That Costs $2.8 Million