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STAT+: Baby KJ scientists hit speed bump in quest to scale custom gene editor
Stat News|3/31/2026
RD
rare diseases
34 stories tracked
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2026-03-052026-03-31
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Stat News17 stories
Stories (Latest First)
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STAT+: FDA approves Rocket gene therapy for rare immune disorder
Stat News|3/27/2026
LINDYsurvived 9 days
STAT+: FDA approves Denali Therapeutics drug for Hunter syndrome
Stat News|3/25/2026
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STAT+: Sarepta Therapeutics shares rise on early promise for rare disease drugs
Stat News|3/25/2026
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STAT+: Six drugmakers, six months: Charting the FDA’s course in the post-Prasad era
Stat News|3/19/2026
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The FDA, urged to avoid controversy, creates a new headache with attack against UniQure
Stat News|3/6/2026
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STAT+: The extremism of the FDA’s Marks and Prasad has come with costs
Stat News|3/5/2026
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STAT+: Prime Medicine to seek approval for gene editing treatment after two-patient trial
Stat News|3/3/2026
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STAT+: UniQure plans to seek approval for Huntington’s therapy still blocked by FDA
Stat News|3/2/2026
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Opinion: I’m a rare disease mom, and I finally have new hope for my son’s future
Stat News|2/27/2026
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STAT+: A rare disease drug was approvable, then it wasn’t. Inside a surprise rejection by the FDA
Stat News|2/25/2026
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STAT+: FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications
Stat News|2/23/2026
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STAT+: BridgeBio drug for genetic cause of dwarfism succeeds in key study
Stat News|2/12/2026
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Opinion: My granddaughter has a rare disease. But clinical trials often exclude patients like her
Stat News|2/11/2026
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STAT+: FDA’s new ‘plausible mechanism pathway’ for personalized gene editing raises concerns
Stat News|1/26/2026