XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

Drug Discovery World

MARCH 2, 2023

New understanding of epigenetics It has been known for more than 20 years that the enzymes placing H3K4me3, a chemical tag added to DNA, are crucial for normal cell development, as well as being linked to leukaemia, breast, bowel and pancreatic cancers.

Gene Expression 52

Gene Expression Gene DNA RNA 52

Drug Discovery World

JANUARY 30, 2024

He said: “The industry, as a whole, needs to carefully consider the true value of curing lifelong genetic or degenerative diseases, particularly when evaluating novel therapeutics that present unique treatment opportunities.” Developing value-based payment models that consider long-term benefits could improve access.

Gene Therapy 72

Gene Therapy Manufacturing Gene Clinical Trials 72

pharmaphorum

JUNE 2, 2022

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. As covered in a previous article , the importance of R&D to develop treatments for rare diseases is high. It is estimated that between 3.5 The price is right.

Drugs 52

Drugs Sales Marketing Development 52

Drug Discovery World

MAY 22, 2023

But are these price tags a barrier to access? In the case of Hemgenix, the assurance behind the price point is that the drug would reduce long-term healthcare costs as patients would have fewer healthcare incidents and need fewer clotting treatments. million per dose.

Gene Therapy 52

Gene Therapy Gene Drugs Contamination 52

The Pharma Data

MARCH 23, 2022

Unfortunately, in a Phase 3 clinical trial, treatment with remdesivir did not make a difference in mortality for Ebola virus disease patients. This special enzyme, once fused to Ebola virus polymerase, enables the polymerase to add a molecular tag to any other protein it interacts with.

Protein 52

Protein Genome Genomics Research 52

Rethinking Clinical Trials

JANUARY 23, 2024

RCT design will vary depending on which CAD manifestation(s) are reflected in the information provided by the imaging test being studied, which affects the treatment target(s) being evaluated in a therapeutic trial. Tags #pctGR, @Collaboratory1 The post Grand Rounds January 19, 2024: Why Are Imaging RCTs Different?

Trials 152

Trials Cardiology Clinical Trials Clinical Trials 152

pharmaphorum

NOVEMBER 16, 2022

The advancements made in drug development over the last decade have seen the arrival of a number of treatments that offer one-time cures and more effective therapies for conditions in smaller patient populations. Advanced therapies have changed the treatment landscape for the industry and for patients. Changing the model.

Gene Therapy 130

Gene Therapy Gene Marketing Development 130

Delveinsight

JULY 22, 2020

Since the data is collected for a long period of time, the doctor can easily evaluate the current and past medical conditions. Since the data is collected over a very long period, it can bring new insight and information which will help understand the functioning of the body and also ensures better medical treatments and productivity.

Doctors 59

Doctors Doctor Marketing Production 59

XTalks

SEPTEMBER 27, 2022

Skysona received FDA approval for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD). About half of patients who do not receive any treatment die within five years of symptom onset. The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it.

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

pharmaphorum

FEBRUARY 16, 2021

There is a curative treatment for people who rely on blood transfusions to survive and maintain their levels of red blood cells. In this first draft guidance NICE raised a series of issues with Zynteglo, which bluebird has already agreed to supply at a confidential discount from its hefty price tag, which is around €1.57

Gene Therapy 91

Gene Therapy Gene Medicine Development 91

XTalks

APRIL 29, 2022

Tirzepatide is a novel investigational obesity treatment that contains mimetics of two hormones that are involved in regulating appetite in a single peptide: a GIP (glucose-dependent insulinotropic polypeptide) receptor agonist and a GLP-1 (glucagon-like peptide-1) receptor agonist. GLP-1 agonists were first developed as diabetes treatments.

Trials 98

Trials Drugs FDA Approval Clinical Trials 98

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Zynteglo’s hefty price tag of $2.8

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

JANUARY 19, 2024

Radiobiological and Clinical Considerations from CRO and Emerging Biotech Radiopharmaceuticals are medicinal formulations containing radioisotopes that are used in the field of nuclear medicine primarily for the diagnosis and treatment of various diseases, particularly cancer.

DNA 66

DNA Radiology FDA Approval Development 66

XTalks

DECEMBER 21, 2022

Additionally, advanced technologies like artificial intelligence (AI) and the adoption of digital tools in healthcare are allowing for improved treatments and care, and powerful research insights. Some experts say the high cost of gene therapies is justified as many are single-dose treatments with curative intent.

Life Science 52

Life Science Gene Therapy Gene Clinical Trials 52

Intouch Solutions

JULY 27, 2021

they pointed out that the COVID-19 vaccines, and the controversial approval of aducanumab for the treatment of Alzheimer’s disease, represented polar opposites in terms of drug pricing reform. In their letter, “ Drug Pricing Reform in 2021 – Going Big or Going Bipartisan? In the case of the vaccines, the U.S.

Drugs 52

Drugs Marketing Vaccination Vaccine 52

Drug Discovery World

NOVEMBER 9, 2023

The luncheon presentation will be given by Hans Gerstmans, Postdoctoral Researcher, Laboratory for Biomolecular Discovery & Engineering, KU Leuven, VIB, alongside Wai Long Tam, Head of Technology Watch at VIB, Technology Watch, VIB.

Engineer 52

Engineer Antibody Protein In-Vivo 52

The Pharma Data

MAY 15, 2021

It records where you’ve been… How long you’ve walked for… How many steps you’ve taken… How many calories you’ve burned… How well you’re sleeping and so on. It automatically detects when you’ve been inactive for too long and gently reminds you to start moving (just like a fitness coach would).

Production 52

Production Containment Medicine Vaccination 52

The Pharma Data

MAY 4, 2021

and developed Europe following the approvals in 2019 for combinations of certain immune checkpoint inhibitors and Inlyta for the first-line treatment of patients with advanced renal cell carcinoma; as well as. Patent Term Extension certificate extending the term of U.S. RECENT NOTABLE DEVELOPMENTS (Since February 2, 2021).

Vaccination 40

Vaccination Vaccine Production Sales 40

Pharmaceutical Technology

NOVEMBER 30, 2022

In October, the US FDA granted Eli Lilly’s Mounjaro (tirzepatide) a Fast Track designation for its use as a treatment for obesity. Several other treatments, like Novo Nordisk’s CagriSema (semaglutide + cagrilintide), are expected to follow after showing encouraging Phase II data. million in Q3. compared to a 5.1% Virgin Islands, U.S.

Drugs 264

Drugs Manufacturing Hormones Contract Manufacturing 264

Pharmaceutical Technology

NOVEMBER 30, 2022

In October, the US FDA granted Eli Lilly’s Mounjaro (tirzepatide) a Fast Track designation for its use as a treatment for obesity. Several other treatments, like Novo Nordisk’s CagriSema (semaglutide + cagrilintide), are expected to follow after showing encouraging Phase II data. million in Q3. compared to a 5.1% Virgin Islands, U.S.

Drugs 246

Drugs Manufacturing Hormones Contract Manufacturing 246

Pharma Marketing Network

JUNE 8, 2020

I mean probably the only real exception is in specialty pharma in terms of, underlying cost actually having gone up. At best, it masks short-term pain while the underlying problem exists. Dave (HR): Oh, no, I think the Specialty Pharmacy. So yeah, there is plenty to talk about there. Don (PMN): Sure.

HR 40

HR Marketing Doctors Doctor 40