NIH and NIDCR Advance First-in-Human Trial for Ultra-Rare Jansen's Disease Therapy
NIDCR prepares a first-in-human trial for Jansen’s disease, advancing a potential therapy for this ultra-rare skeletal disorder through NIH collaboration.
Read the full articleYou might also wanna read
ABPI welcomes rare diseases research landscape report
The National Institute for Health and Care Research (NIHR) has today published the first Rare Diseases Research Landscape report for the UK.
Neurology Journal: Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Dr. Crowell interviews Dr. Timothy Yu about his article "Patient-customized oligonucleotide therapy for a rare genetic disease." Read the NE
Prion Disease Clinical Trial NN112
Dr. Gregg Day and Drs. Sonia Vallabh and Eric Minikel discuss scientific insights and the future of prion disease treatment, highlighting th

Neutrolis Announces New England Journal of Medicine Publication Demonstrating First Clinical Evidence for NET-Degrading Therapy in Severe, Treatment-Refractory Systemic Lupus Erythematosus (SLE) Caused by DNASE1L3 Deficiency
Treatment with investigational DNASE1L3 analog NTR-441, which degrades neutrophil extracellular traps (NETs), resulted in rapid clinical imp
Medical student helps research rare neurologic condition clustered in Dominican Republic
Alfred "Aj" Espinoza (photo by Jim Howe) Alfred "Aj" Espinoza (photo by Jim Howe) A rare neurologic condition called pantothenate kinase-ass

VNDA Stock Gains After-Hours As FDA Grants Rare Pediatric Disease Designation For Experimental Therapy
The designation covers VCA-894A, Vanda’s investigational treatment for Charcot-Marie-Tooth disease type 2S.

Comments
Sign in to join the conversation.
No comments yet. Be the first.