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 ...
Targeted Oncology16h
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 ...
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 ...
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 — ...
ophthalmologytimes8d
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 ...
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 ...
The Pharma Letter15d
FDA grants rare pediatric status for Cartesian’s Descartes-08
Cartesian Therapeutics today announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease ...
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 ...