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

FDA Insurance 115

pharmaphorum

JANUARY 21, 2022

“Access to medicines remains one of the most challenging policy areas in every country we work with through the pan-European region,” said Natasha Muscat, Director of Country Health Policies and Systems at World Health Organization (WHO) Europe. Flawed funding model.

Insurance 52

Insurance Vaccines 52

pharmaphorum

AUGUST 10, 2020

Evrysdi is the third treatment approved for SMA , an ultra-rare muscle wasting disease that can begin in early childhood, after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene abeparvovec). Evrysdi also improved survival without permanent ventilation at 12 and 23 months, compared to natural history.

FDA 57

FDA Specialty Pharmacies 57

pharmaphorum

NOVEMBER 28, 2022

Owkin is simultaneously releasing two other open-source projects: FLamby, a federated learning model-ready dataset that researchers can use to develop models before training them on other data sets, and SecureFedYJ, a tool for normalising real-world healthcare data while maintaining data privacy.

Hospitals 70

Hospitals 70

The Physician Pharmacist

SEPTEMBER 14, 2023

I think a lot of physicians would be lost without their pharmacist on call in a lot of hospitals. It's very nitpicky, it's very detailed and it's very disease or it's very does disease oriented, I'll say for lack of a better term. But that that really I would say is the is the only true difference in practice.

Medical Schools 52

Medical Schools Hospitals Communication Patient Care 52

European Pharmaceutical Review

DECEMBER 27, 2023

Bridging therapy has been shown to correlate with an inferior response to CAR T-cell therapy due to both progression of the chemotherapy-refractory malignant disease during the manufacturing period and increased toxicity. This is in addition to its considerable price tag. New England Journal of Medicine. 2013;368(16):1509–18.

Adverse Reactions 87

Adverse Reactions Immunization Medical Schools Chemotherapy 87

Pharmaceutical Technology

NOVEMBER 30, 2022

In 1997, the World Health Organization (WHO) classified obesity as a chronic disease. High rates of obesity place a major burden on healthcare systems as the condition is associated with many major health complications like type 2 diabetes, hypertension, non-alcoholic fatty liver disease, and other bodily dysfunctions.

Insurance 105

Insurance Labelling Communication FDA 105