MDA Kickstart Program MDA Kickstart Program Receives FDA Rare Pediatric Disease Designation and Orphan Drug Designation ...
A TEEN has spent the last 10 years in a wheelchair after he was diagnosed with a rare genetic muscular disease known as ...
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At #STATSummit, three parents discussed the challenges they've faced trying to find treatments for their children with rare ...
This is the first head-to-head trial on this rare muscle disease and the results will directly inform patient care and provide more 'real-world' options for patients." This research will impact ...
Doha, Qatar: Sidra Medicine's Genetic and Genomic medicine clinic is providing life-altering gene therapy for rare genetic ...
Researchers found that ultra-rare genetic variants play a significant role in the heritability of coronary artery disease ...
Spinal muscular atrophy (SMA) is a rare genetic disorder that causes muscle weakness and disability, typically in young ...
Home invasive mechanical ventilation (HIMV) for chronic respiratory insufficiency is rare in Finland, where extended NIV is now preferred.
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