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 ...
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 ...
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 ~~ ...
BioSpace2d
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 ...
Fierce Pharma1d
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 ...
CURE14d
FDA Grants Rare Pediatric Designation to Treatment With NEO100 in Brain Cancer
The FDA has granted Rare Pediatric Disease Designation for NEO100 in the treatment of pediatric-type diffuse high-grade gliomas, a type of brain cancer.
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 ...
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 ...
Odylia Therapeutics drives commitment to rare disease drug development through Rare Disease Day event
Industry leaders gather to discuss innovative solutions for accelerating the development of therapeuticsBOSTON, MA, April 10, ...
BioPharma Dive2d
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 ...
WebMD29d
FDA OKs First Treatment for Rare Kidney Disease
March 21, 2025 – The FDA has approved a drug as the ... the approval marks the first treatment that targets the cause of the disease. C3G happens when an overactive immune system causes a ...