Rethinking Clinical Trials

OCTOBER 26, 2023

Untreated, chronic Hepatitis C infection leads to liver damage, liver cancer, and death. show that a cure is not sufficient to guarantee disease elimination. with the rate of reported acute Hepatitis C cases increasing 400% during 2010-2020. Rates are the highest among 20-39-year-olds.

Clinical Research 147

Clinical Research Clinical Trials Clinical Trials Research 147

pharmaphorum

JANUARY 9, 2023

” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 ” NHS England said the temporary change in eligibility – which ties in with NICE guidance – has been implemented as there are two other disease-modifying drugs available on the NHS for the treatment of SMA.

Gene Therapy 114

Gene Therapy Genetics Gene Clinical Trials 114

XTalks

JANUARY 28, 2022

Up to 50 percent of patients will develop metastatic disease, mainly spreading to the liver, making treatment difficult and limited. The new treatment will thus help address the unmet need for the disease. Kimmtrak has a price tag of $18,760 per vial, which amounts to a weekly dose.

FDA Approval 98

FDA Approval Development Licensing Licensing 98

XTalks

OCTOBER 26, 2021

The treatment came with an initial price tag of $39,000 per vial, which amounts to a total of $575,00 each year. A lack of the gene leads to accumulation of porphyrins — essential compounds found in hemoglobin — in the liver. Two sisters living in the UK with AIP described their experience having the disease in a BBC article.

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

XTalks

NOVEMBER 24, 2022

The adeno-associated virus vector-based gene therapy delivers a functional copy of Factor IX, a protein involved in blood clotting that is missing or not made in enough amounts in patients who have the disease due to mutations in the gene that codes for it. Hemophilia B primarily affects men.

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetics 52

XTalks

OCTOBER 12, 2022

Features of blood perfusion in the brain detected by MRI now have clinical applications in central nervous system (CNS) cancers, neurodegenerative disease, cerebrovascular disease and demyelinating disease. DCE-US can be done for the liver and for the abdomen without too much trouble,” added Ashton.

Clinical Trials 93

Clinical Trials Clinical Trials Trials Drugs 93

pharmaphorum

FEBRUARY 22, 2021

Last week, Bluebird said it was pausing its Zynteglo (betibeglogene autotemcel) programme in Europe after a phase 1/2 trial in patients with sickle cell disease (SCD) – another red blood cell disorder – turned up two cases of blood cancers, one of acute myeloid leukaemia (AML) and another of myelodysplastic syndrome (MDS). million in Europe.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52