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.

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pharmaphorum

AUGUST 18, 2022

.” The gene therapy specialist’s chief executive Andrew Obenshain said the approval “marks a watershed moment for the field of gene therapy” and is “ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care.”

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pharmaphorum

JANUARY 31, 2023

Given the high price tags placed on many medications, it’s easy to understand why patients often feel that they are being taken advantage of. Finally, the belief that pharmaceutical companies prioritise their bottom lines over the health and well-being of patients is probably the largest contributing factor to lack of trust in the industry.

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pharmaphorum

AUGUST 26, 2022

BioMarin will no doubt have an eye on the earlier experience of bluebird bio, which secured EU approval for rare disease gene therapy Zynteglo (betibeglogene autotemcel) in 2019 for beta thalassaemia with a price tag of around €1.6 million spread over five years.

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Evolve Your Success

JULY 19, 2022

One of the key million-dollar tips here is to tag the person in the first sentence, “@So-and-so, that’s a great point.” Other surgeons don’t get replied back because they don’t tag the poster or the person who posted. Social Selling: Sometimes when you do post, you need to go back with a critical eye.

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Cadensee

JANUARY 1, 2022

So basically, we allow imaging of the throat, the ear, the skin, the eyes as quotation; recording of the sounds of the heart, the lungs, the stomach. We are today supporting chronic management at the family level for respiratory disease and cardiac. And basic vital signs like heart rate, temperature reading, spirometry and so forth.

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