US FDA approves IntraBio's drug for rare genetic disease
The U.S. Food and Drug Administration approved IntraBio's drug for a rare and fatal genetic disorder, the health regulator ...
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FDA OKs First Treatment for Rare, Deadly Genetic Disorder
Miplyffa treats Niemann-Pick disease type C, which progressively impacts the abilities to speak, swallow, and walk or move ...
Researchers identify genetic mutations for rare disorder causing cognitive decline
A rare cause of hereditary cognitive decline known as CSF1R-Related Disorder (CSF1R-RD) gets its name from mutations in the ...
Sutro Biopharma's Competitive ADCs Position It For Future Growth In Oncology
Sutro Biopharma specializes in ADCs for oncology and shows promise with STRO-002 for ovarian cancer and collaborations with ...
Lantern Pharma Gets Three FDA Rare-Pediatric Designations for LP-184
Lantern Pharma has won a trio of rare-pediatric-disease designations from the Food and Drug Administration for its investigational drug candidate LP-184 in multiple ultra-rare children's cancers.
TMCnet1d
Lantern Pharma Announces Three U.S. FDA Rare Pediatric Disease Designations Granted to LP-184 in Multiple Ultra Rare Children's Cancers
Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence ("AI") company developing targeted and transformative cancer therapies using its proprietary RADR ® AI and machine learning ("ML") ...
GEN1d
StockWatch: FDA Surprise Lifts Expectations for Applied’s Govorestat
Investors and analysts predict the FDA will approve Applied Therapeutics' lead candidate govorestat for classic galactosemia ...
Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive ...
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Longboard Stock Soars on FDA Designations for Epilepsy Drug
Shares of Longboard Pharmaceuticals LBPH gained 14% on Thursday after it announced that the FDA granted two designations — ...
FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C — 2nd Update
Zevra Therapeutics has won Food and Drug Administration approval of Miplyffa for Niemann-Pick disease type C, or NPC, making it the first drug cleared in the U.S. for the rare neurodegenerative ...
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Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...
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House E&C Committee Passes Bill on Rare Pediatric Disease Drug Development
The bill's passage through the committee improves the rare pediatric disease priority review voucher program's chances of being reauthorized before it expires Sept. 30.