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Stealth scores FDA approval for 1st Barth syndrome treatment

Stealth’s injection will be sold as Forzinity for Barth syndrome, an X-linked genetic disorder that weakens the heart and other muscles.
Gabrielle Masson Sep 19, 2025 6:00pm
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Daiichi, Merck rev up for accelerated approval filing

Sep 7, 2025 10:45am
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Regenxbio's Hunter gene therapy latest to face FDA delay

Aug 19, 2025 10:30am
Fabry disease

Cash-strapped Sangamo seeks partner for new gene therapy

Jun 24, 2025 11:53am
rare diseases

Stealth CEO questions FDA rejection of rare disease drug

May 29, 2025 2:27pm
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Regenxbio starts pivotal study for DMD gene therapy

Nov 18, 2024 2:24pm