Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-
AstraZeneca has added a late-stage rare disease therapy to its pipeline, buying France’s Amolyt Pharma and its hypoparathyroidism drug eneboparatide for $800 million upfro
NICE has turned down Sanofi’s Xenpozyme as a treatment for the rare disease acid sphingomyelinase deficiency (ASMD), creating a situation where access to the drug in the U
Avidity Biosciences has filed a private placement worth around $400 million, seeking a cash infusion to help advance its pipeline of clinical-stage candidates that combine
Rare Disease Day saw the debut of a film that aims to raise awareness of cerebral adrenoleukodystrophy (cALD), a devastating and fatal condition with no pharmacological tr
Almost four years after it was approved in Europe to treat the incurable rare disease systemic amyloid light-chain (AL) amyloidosis, Johnson & Johnson’s Darzalex has b
Endeavor BioMedicines has completed a third-round financing, raising an impressive $132.5 million to fund trials of its two clinical-stage drug candidates for pulmonary fi
It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.