Targeted Oncology1h
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, ...
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 ...
Capricor: DMD Treatment Focus With Deramiocel Is Likely To Pay Off
Capricor announced it intends to file a BLA for deramiocel for treatment of patients with DMD cardiomyopathy. Click here to ...
FDA backs Somite.ai's new drug for rare children's muscle disease
AI-driven biotech Somite.ai scores dual FDA designations for its novel treatment, potentially fast-tracking hope for families ...
STAT12d
A rare disease program, portrayed as both lifeline and pharma handout, is set to expire
Advocates for people with rare diseases say a government voucher program has been crucial to pursuing treatments. That ...
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 ...
Zacks.com on MSN4d
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 — ...
ophthalmologytimes7d
Atsena Therapeutics receives Orphan Drug Designation from FDA for ATSN-201 gene therapy to treat X-linked retinoschisis
The announcement marks the second FDA designation for ATSN-201, which previously received Rare Pediatric Disease designation.
Cartesian Therapeutics Receives FDA Rare Pediatric Disease Designation for Descartes-08 for the Treatment of Juvenile Dermatomyositis
Descartes-08, Cartesian’s lead mRNA cell therapy candidate, is an autologous mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T) product candidate targeting B-cell maturation antigen ...
Muscular Dystrophy News5d
DMD treatment SMT-M01 gets FDA orphan drug, rare disease tags
DMD treatment SMT-01, a cell replacement therapy, received FDA orphan drug and rare pediatric disease designations.
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 ...