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 ...
WebMD6h
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 ...
Targeted Oncology6h
LP-184 Earns 3 FDA Rare Pediatric Disease Designations in Solid Tumors
The novel agent LP-184 has been granted rare pediatric disease designations in malignant rhabdoid tumors, rhabdomyosarcoma, ...
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.
Le Lézard1d
Immusoft to Announce Positive Phase 1 Data for First Engineered B Cell Therapy in a Clinical Trial
Immusoft of CA, a wholly owned subsidiary of Immusoft Corporation ("Immusoft"), a cell therapy company dedicated ...
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 ...
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 ...
Kazia Therapeutics Announces Presentation of EVT801 Clinical Data at 15th Biennial Ovarian Cancer Research Symposium
Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, is pleased to announce the ...
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 ...
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 ...