pharmaphorum

JANUARY 18, 2023

The DNA of these so-called ‘genetic superheroes’ may contain clues about how to treat severe disease. In our experience of running 20+ precision medicine studies, there are a few design principles that every research programme – from biobanks to clinical trials – should consider. Giving participants something in return.

Genetic Disease 97

Genetic Disease Genetics Research Genome 97

XTalks

APRIL 24, 2023

Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases. Reference: Chung DC, et al.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Delveinsight

DECEMBER 13, 2020

Even though clinical trial results were promising, adverse events and serious complications were observed. Hepatitis C treatment options, without a doubt, have improved significantly, including pan-genotypic medications that are used to treat all genotypes and subtypes. and GT2 and GT3 with ribavirin. in January 2019.

Genotype 52

Genotype FDA Approval DNA Drugs 52

The Pharma Data

NOVEMBER 2, 2020

(NASDAQ:ADIL; ADILW) , a clinical-stage biopharmaceutical company focused on the development of treatments for addictions, today provided an update on its landmark ONWARD Phase 3 pivotal trial. 40% of expected Trial patient screenings have been completed and the Trial is more than 25% enrolled relative to planned enrollment.

Trials 52

Trials Genotype Marketing Production 52

XTalks

MARCH 3, 2021

Molybdopterins are a class of cofactors found in most molybdenum-containing and all tungsten-containing enzymes. The efficacy of Nulibry in the treatment of MoCD Type A was demonstrated in data from three different clinical trials that was compared to data from a natural history study.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

SEPTEMBER 15, 2020

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. So, this really validates what we already knew and now we have prospective results supporting that [from this trial].”.

Vaccination 103

Vaccination Vaccine Insulin Trials 103

XTalks

APRIL 15, 2021

Late-stage clinical trial failures are largely to blame for the lack of effective treatment options for patients with HFpEF, and poor five-year survival rates make this an area of great unmet need. A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl.

Development 98

Development Drugs Genome Genomics 98