NorthSea Therapeutics bags FDA rare paediatric disease tag for NASH drug
Pharmaceutical Technology
OCTOBER 17, 2023
The Netherlands-based company’s treatment has been awarded the designation by the US FDA following a successful Phase I trial.
Pharmaceutical Technology
OCTOBER 17, 2023
The Netherlands-based company’s treatment has been awarded the designation by the US FDA following a successful Phase I trial.
pharmaphorum
MARCH 20, 2024
Orchard Therapeutics has revealed the US price of Lenmeldy, its gene therapy for rare disease MLD, placing a $4.25m price tag on the one-shot treatment
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Pharmaceutical Technology
SEPTEMBER 30, 2022
Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.
Pharmaceutical Commerce
MARCH 6, 2024
Navigating the complex landscape of healthcare coverage can be an intimidating task, especially for patients with rare diseases for which treatments often come with a high price tag.
Pharmaceutical Technology
JUNE 29, 2022
This year has already been eventful when it comes to the development of therapies for rare diseases. Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. Prior to the program, only 10 drugs were approved for a rare disease.
XTalks
FEBRUARY 27, 2024
Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy.
Rethinking Clinical Trials
JANUARY 23, 2024
People with angina-like symptoms are often not patients with a disease. Most do not have obstructive coronary artery disease (CAD), but a few are very high risk. Costs are rarely a significant factor in comparing different testing approaches. They are trying to follow the guidelines for symptoms and disease.
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