Elixirgen Therapeutics Receives Rare Pediatric Disease Designation for EXG-34217 in Dyskeratosis Congenita and Related Telomere Biology Disorders
Elixirgen Therapeutics is currently enrolling patients in its Phase 1/2 trial evaluating EXG-34217 in patients with telomere biology disorders at Cincinnati Children’s Hospital Medical CenterBALTIMORE ...
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 ...
Business Wire5d
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 ...
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.
Business Wire6d
Longboard Pharmaceuticals Receives Rare Pediatric Disease Designation and Orphan Drug Designation for Bexicaserin (LP352) in Dravet Syndrome
“We are thrilled to receive both Rare Pediatric Disease and Orphan Drug designations for bexicaserin in Dravet syndrome. As the first company to receive Breakthrough Therapy designation ...
Stockhouse5d
Longboard Pharmaceuticals Receives Rare Pediatric Disease Designation and Orphan Drug Designation for Bexicaserin (LP352) in Dravet Syndrome
Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that the FDA ...
Neu-REFIX Beta Glucan Receives Rare Pediatric Disease And Orphan Drug Designations From The US FDA For Treatment Of Duch...
The Neu-REFIX Beta 1,3-1,6 glucan, is an exo-polysaccharide produced by N-163 strain of Aureobasidium Pullulans, available as ...
Local life sciences firms fear loss of big-money FDA voucher exchange
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Financial Post5d
Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
made in Japan has received both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (US FDA) for treatment of Duchenne muscular ...
AFP6d
Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
Neu REFIX Beta glucan, made in Japan has received both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (US FDA) for treatment ...
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 ...