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Maroc Maroc - ZACKS.COM - All Stories - 01/Jul 14:31

FDA Rejects Rocket's (RCKT) Gene Therapy BLA for Rare Disease

Per the FDA, Rocket (RCKT) needs to submit additional manufacturing information on Kresladi gene therapy to secure approval for severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder.

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Rocket Pharmaceuticals Provides Regulatory Update on Kresladi (marnetegragene autotemcel; marne-cel)

drugs.com - 28/Jun 05:07

CRANBURY, N.J.--(BUSINESS WIRE)--Jun. 28, 2024-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company...

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FDA Expands Approval for Duchenne Muscular Dystrophy Gene Therapy

drugs.com - 24/Jun 21:21

MONDAY, June 24, 2024 -- The U.S. Food and Drug Administration has expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene...

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Sarepta Therapeutics Announces Expanded US FDA Approval of Elevidys to Duchenne Muscular Dystrophy Patients Ages 4 and Above

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 20, 2024-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare...

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Chinese space rocket’s accidental take-off results in fiery conclusion

smh.com.au - 01/Jul 04:24

Falling rocket debris in China after launches is not unheard of, but it is very rare for part of a rocket under development to make an unplanned...

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Chinese space rocket’s accidental take-off results in fiery conclusion

smh.com.au - 01/Jul 04:24

Falling rocket debris in China after launches is not unheard of, but it is very rare for part of a rocket under development to make an unplanned...

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A Rare Genetic Variant Could Delay Alzheimer’s, Study Shows

greekreporter.com - 23/Jun 16:00

In 2019, scientists identified a Colombian woman who, as a result of her genes, should have experienced symptoms of Alzheimer’s disease starting in...

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Eli Lilly (LLY) Gets FDA Nod for Alzheimer's Drug Donanemab

zacks.com - 03/Jul 12:34

Eli Lilly's (LLY) Alzheimer's disease drug, Kisunla (donanemab), wins FDA approval. Kisunla will be the second drug on the market to treat the...

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Patritumab Deruxtecan BLA Submission Receives Complete Response Letter from FDA Due to Inspection Findings at Third-Party Manufacturer

drugs.com - 26/Jun 04:06

BASKING RIDGE, N.J. & RAHWAY, N.J., June 26, 2024 – The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL)...

Test Identifies Rare Fatal Genetic Heart Condition

medindia.net - 21/Jun 09:50

New clinical test detects Calcium Release Deficiency Syndrome (CRDS), a genetic arrhythmia with rapid heartbeats leading to sudden cardiac arrest.