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.

FDA 115

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pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

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Pharmaceutical Technology

MAY 2, 2023

While initially only approved for metastatic disease, ICIs have now moved into earlier disease settings, reducing the risk of disease progression and relapse. All currently approved CAR-Ts are autologous, with the patient’s T-cells being genetically engineered to target antigens expressed by the cancerous cells.

Physicians 52

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Celeritas

NOVEMBER 7, 2022

In conjunction with tags, facial expression readers, or electromagnetic RFID tags, drones can monitor the health indicators of individual herd members. DES can be used on a livestock farm to identify the events affecting the fluctuation of yields such as lactation stage, weather events, diseases, or feeding practices.

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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.

Medicine 59

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pharmaphorum

DECEMBER 23, 2022

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Add to this the considerable cost of these medications – the first approved CAR-T, Novartis’ Kymriah (tisagenlecleucel), had a U.S. CellCentric, a U.K-based

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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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