News

Ultragenyx: Commercial Engine Fuels Rare Disease Pipeline
Ultragenyx's strong revenue growth, diverse streams, and late-stage pipeline make it a standout in rare disease and gene ...
Ascendis Pharma: Rare Disease Player Is A Buy On Yorvipath Launch Trajectory
ASND thrives amid market volatility, with Yorvipath's strong potential & strategic focus on rare diseases. Click here to find ...
Eric Dane reveals ALS diagnosis: What to know about the rare disease
Eric Dane isn't letting his health challenges slow him down. The "Grey's Anatomy" alum, 52, revealed he's been ...
STAT1d
I don’t know what I’m supposed to call my rare kidney disease
I care deeply because this chronic disease is mine. It lives inside me. It’s radically changed my life and the life of my ...
Patients of rare disease welcome prospect of dramatic slash in price of drug
Parents of children with spinal muscular atrophy (SMA), a rare disease, welcomed the announcement by the Indian pharma ...
Fierce Pharma2d
Ipsen's liver disease drug Iqirvo off to promising launch in battle with Gilead's Livdelzi
Ipsen and its fellow French partner Genfit are competing with Intercept and Advanz’s Ocaliva, which was approved in 2016 and ...
BioSpace3d
Glycomine Gets $115M Series C for Rare Disease, Aims for Phase II Launch Mid-Year
The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a ...
FDA Approval of Amgen's UPLIZNA for IgG4-related Disease Opens New Market Opportunity in Rare Autoimmune Segment | DelveInsight
IgG4-related disease (IgG4-RD) is a systemic fibroinflammatory condition marked by the accumulation of IgG4-positive plasma ...
uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington’s Disease
Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics ...
Proof-of-Concept Data for Rare Disease Indications MVID and SBS-IF for Jaguar Health's Crofelemer to be Presented at April 2025 ELITE PED-GI Congress
Crofelemer, a novel plant-based anti-secretory prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines ...
BioPharma Dive3d
Startup Glycomine raises $115M to push rare disease drug deeper into testing
The biotech is developing an experimental drug for the most common of a family of disorders all caused by errors in ...
BioPharma Dive5d
Translating FDA guidance into action: Regulatory considerations for orphan drug developers
Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than ...