FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency
The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the ...
FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...
Daily3d
FDA Approves First Gene Therapy for Treatment of AADC deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
IND for ATA-200, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5), cleared to proceed by FDA
EVRY, France, November 12, 2024--"This IND clearance in an important step to bring ATA-200 to US children suffering from this highly debilitating LGMD-2C/R5 disease" ...
eLife6d
Endophilin A1 facilitates organization of the GABAergic postsynaptic machinery to maintain excitation-inhibition balance
This study presents a valuable finding on the molecular mechanisms that govern GABAergic inhibitory synapse function. The authors propose that Endophilin A1 serves as a novel regulator of GABAergic ...
precisionmedicineonline18h
Neurogene Halts Testing High-Dose Rett Syndrome Gene Therapy After Adverse Event
Following this update, the company's stock price dropped more than 30 percent from market close Friday to $23.79 per share Monday morning.
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A Beginner's Guide To Greenwash And Four Ways To Avoid Falling For It
Food scraps were fed to our cattle ... Askhat Gilyakhov / Alamy Stock Vector , CC BY-NC-ND Plastic recycling labels can be confusing too. The triangle with three chasing arrows, called the Mobius Loop ...
4DMT Reports Third Quarter 2024 Financial Results, Operational Highlights and Expected Upcoming Milestones
Presented positive interim data for 4D-150 in wet age-related macular degeneration (wet AMD) from PRISM Phase 1/2 clinical trial highlighting ...
AZoOptics on MSN6d
Using FT-NIR and Machine Learning for Identifying Authenticate Kimchi
By using the near-infrared region of the electromagnetic spectrum, this method can reveal a sample's chemical composition ...
precisionmedicineonline7d
Neurogene Dropping Batten Disease Gene Therapy; Discloses Adverse Event in Rett Syndrome Trial
The New York-based company said it applied for regenerative medicine advanced therapy designation with the US Food and Drug ... in an ongoing open-label Phase I/II trial. NGN-401 uses an ...
PTC Therapeutics, Inc.: PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - ...
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Direct Harm stock illustrations
Recycle mark battery trash can NG image icon Recycle mark battery trash can NG image icon direct harm stock illustrations Recycle mark battery trash can NG image icon Sodium free food label vector ...