tag orphan-diseases 
STAT
APRIL 12, 2024
In response to controversy, a group of Colorado lawmakers is abandoning a bill that would have required a new state board — which is chartered with setting caps for the cost of medicines — to create exemptions for rare disease drugs.
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. Most, if not all, of these therapies used the FDA’s Orphan Drug Designation to aid their development plans.
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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. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.
Pharmaceutical Technology
JULY 14, 2022
It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Go-to-market strategies.
PharmaShots
JANUARY 27, 2023
Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)
pharmaphorum
JUNE 2, 2022
R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. As covered in a previous article , the importance of R&D to develop treatments for rare diseases is high. of the global population are affected by a rare disease.
The FDA Law Blog
JUNE 27, 2023
Amongst his accomplishments, Law360 considered the role James has played in leveraging little-used pathways to FDA approval for often first-ever drugs to treat rare diseases (e.g., Duchenne, Friederichs’s Ataxia, Sickle Cell Disease, Chagas, ALS).
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