XTalks

APRIL 24, 2023

Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. Terence Eagleton, MB BS, Senior Medical Director at the global clinical research organization (CRO) Medpace. Reference: Chung DC, et al.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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

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

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. Both drugs resulted in inhibition of HCV replication.

Genotype 52

Genotype FDA Approval DNA Drugs 52

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. In the trial, the vaccine was injected directly into the lymph nodes of type 1 diabetic individuals. The DIAGNODE-2 Trial.

Vaccination 103

Vaccination Vaccine Insulin Trials 103

XTalks

MARCH 3, 2021

Molybdopterins are a class of cofactors found in most molybdenum-containing and all tungsten-containing enzymes. Nulibry Trial Results. 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

The Pharma Data

MARCH 25, 2021

Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. SG contains a genotoxic component and can cause teratogenicity and/or embryo-fetal lethality when administered to a pregnant woman. About the ASCENT Study. Embryo-Fetal Toxicity.

Genotoxicity 52

Genotoxicity Genotype Containment Protein 52