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 ...
AI model identifies existing drugs that can be repurposed for treatment of rare diseases
There are more than 7,000 rare and undiagnosed diseases globally. Although each condition occurs in a small number of ...
How do rare genetic variants affect health? AI provides more accurate predictions
Whether we are predisposed to particular diseases depends to a large extent on the countless variants in our genome. However, ...
Neurolixis Secures Orphan Medicinal Product Designation For NLX-112 As A Treatment For Spinocerebellar Ataxia
European Commission approves OMP Designation for NLX-112 (befiradol) following compelling results in studies funded by the US Dept of De ...
US FDA approves IntraBio's drug for rare genetic disease
Niemann-Pick disease type C (NPC) is a rare genetic disorder that affects the nervous system and other organs, causing ...
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.
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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") ...
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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 ...