XTalks

AUGUST 21, 2023

“For the first time, doctors have an approved medicine available to them, shown to reduce the formation of new, abnormal bone growth, known as heterotopic ossification (HO), which causes debilitating mobility challenges and has a devastating impact on the lives of people with FOP.”

Drugs 98

Drugs FDA Approval Genetics Clinical Trials 98

Bioengineer

JULY 22, 2021

Geneticists from Trinity College Dublin have discovered how a specific genetic mutation called H3K27M causes a devastating, incurable childhood cancer, known as diffuse midline glioma (DMG), and – in lab studies working with model cell types – successfully reverse its effects to slow cancer cell growth with a targeted drug.

Genetics 52

Genetics Drugs Clinical Trials Clinical Trials 52

pharmaphorum

SEPTEMBER 21, 2021

Global Gene’s Craig Martin introduces a new patient identification initiative that strives to improve diagnosis and access to clinical trials for all patients with rare CNS conditions. Doctors are often unfamiliar with these conditions. Rare diseases impact more than 30 million Americans, and 400 million people around the globe.

Gene Therapy 90

Gene Therapy Gene Clinical Trials Clinical Trials 90

pharmaphorum

SEPTEMBER 17, 2020

US medical experts are reportedly concerned that a neurological side effect picked up in AstraZeneca’s closely-watched COVID-19 vaccine trial could compromise the whole project, as the FDA weighs whether to give the go ahead for US studies to resume. Feature image courtesy of Rocky Mountain Laboratories/NIH.

Vaccination 105

Vaccination Vaccine Trials Doctors 105

pharmaphorum

MAY 21, 2021

Meanwhile the autologous CAR-Ts already approved and on the market require patients’ T-cells to be harvested and genetically modified to fight cancer, an expensive and time-consuming process. The post Allogene set to disrupt CAR-T market with pivotal trial of ‘off the shelf’ therapy appeared first on.

Marketing 53

Marketing Trials Manufacturing Doctors 53

pharmaphorum

AUGUST 1, 2022

Sarepta reported updated clinical trial results with the one-shot therapy in July which bolstered the data for SRP-9001 (delandistrogene moxeparvovec) for efficacy and durability, but also raised a concern about its safety after a serious case of myocarditis was seen in one of 38 patients enrolled in its ENDEAVOR study.

Gene Therapy 105

Gene Therapy Gene Trials Genetics 105

Pharmaceutical Technology

JUNE 29, 2022

In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Continuing advancements within this field deliver new hope to doctors and patients, transforming disease outcomes for previously incurable indications.

Drugs 295

Drugs Gene Therapy Manufacturing Production 295

STAT News

MARCH 6, 2023

Still, when doctors told her last year that the cancer was growing despite two operations, radiation therapy, and a fifth regimen of chemotherapy, the retired business-meeting facilitator decided to do something unorthodox: spend $83,000 out of pocket on an unproven experimental cancer vaccine.

Vaccination 98

Vaccination Vaccine Drugs Clinical Trials 98

pharmaphorum

JUNE 24, 2022

Last year, AstraZeneca reported that a new drug to treat Wilson disease developed by its Alexion rare diseases unit had hit the mark in a phase 3 trial, setting it on course for regulatory filings. In a statement , AZ said most patients in the trial had low neurological symptom scores at baseline, giving little room to show an improvement.

Drugs 100

Drugs Gene Therapy Compliance Trials 100

Drug Discovery World

OCTOBER 18, 2023

4 Unfortunately, many AAV-based gene therapies fail during clinical trials, despite them appearing safe and efficacious in preclinical studies. For example, researchers may find that an AAV vector-based gene therapy displays excellent transduction efficiency in a preclinical model, only to see the efficiency drop in clinical trials.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

pharmaphorum

DECEMBER 21, 2020

As it’s just a messenger molecule, it does not affect the body’s own genetic code when it is injected as a vaccine – but what it does do is instruct cells to code for copies of a certain protein. They are very good doctors, scientists and entrepreneurs. He told pharmaphorum: “They are very good doctors, scientists and entrepreneurs.

Vaccination 111

Vaccination Vaccine Doctors Doctor 111

The Pharma Data

DECEMBER 17, 2020

Dr. Lisa Forbes — his doctor and co-author of a letter describing his case in the Oct. This patient had a genetic mutation that ruxolitinib is known to work on. Forbes said it’s not clear if other people with type 1 diabetes also have this specific genetic mutation. WEDNESDAY, Oct.

Insulin 52

Insulin Genetics Doctors Doctor 52

The Pharma Data

DECEMBER 11, 2020

And even when Black people with AML have the same access to treatment as white patients, their survival is shorter — something genetic differences might explain. But even when patients of both races received the same treatments, Black patients’ outcomes were worse, clinical trial data showed.

Genetics 52

Genetics Clinical Trials Clinical Trials Genetic Analysis 52

XTalks

JUNE 8, 2021

In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. The prevalence of SMA is about one in 10,000 children, and one in 54 people carry the SM1 genetic mutation; two carriers have a 25 percent chance of having a child with SMA.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

ACRP blog

OCTOBER 6, 2022

population, but comprise over 90% of the population in clinical trials. The total percentage of minority inclusion from all clinical trials in the United States during the last 25 years was just 3.95%. [1]. Study sites may also add any of their clinical trials at no cost. of the U.S. 1] Manuel A. Ma, MD, MAS; Dora E.

Research 98

Research Clinical Trials Clinical Trials Trials 98

pharmaphorum

AUGUST 25, 2022

That was until my doctors stunned me with the news that I had cirrhosis caused by non-alcoholic steatohepatitis, or NASH, a severe form of fatty liver disease that often has few or no symptoms until very late stages. Obesity, diabetes, and genetics are the primary drivers, and COVID is about to make this exponentially worse.

Development 98

Development Drugs Trials Medicine 98

XTalks

APRIL 6, 2022

Therefore, these patients could have a clinical benefit from further treatment or participation in a clinical trial. If that happens, it is a big setback for patients and the doctors treating them,” said Dr. Rosenfeld in a news release by Cancer Research UK. The LUCID study indicated that relapse is likely if ctDNA is detected.

DNA 105

DNA In-Vitro Doctors Doctor 105

pharmaphorum

JANUARY 15, 2023

Depending on a patient’s underlying genetic makeup, it can affect anywhere between 0.4 Yet, the data that Horizon presented demonstrated consistent efficacy in NMOSD patients treated with UPLIZNA, regardless of a particular genetic polymorphism or genetic variability that can diminish responses to other monoclonal antibody therapies.

Antibody 105

Antibody Genetics Medicine Drugs 105

pharmaphorum

AUGUST 13, 2020

It’s bad form to compare performance of drugs in separate clinical trials, but results suggest that Viltepso helps patients produce more dystrophin than Sarepta’s rival Vyondys 53 (golodirsen). Viltepso was evaluated in two clinical studies with a total of 32 patients, all of whom were male and had genetically confirmed DMD.

FDA Approval 52

FDA Approval Drugs Gene Trials 52

pharmaphorum

AUGUST 11, 2022

The participants undergo monitoring for genetic variations that could potentially negatively impact their reaction to therapies. Pharmacogenomics is ushering in an era where we can eliminate those struggling with a trial-and-error system that leaves many getting sicker even when effective treatments exist for them. About the Author.

Genome 98

Genome Genomics Genetics Drugs 98

XTalks

MAY 20, 2022

Doctors and physicians rely on the results provided by a clinical scientist to make an accurate prognosis, diagnosis and assessment for a patient’s treatment plan. Here is a summary of the kind of work you can expect to do: Design and implement clinical trials for human diseases and treatment. Genetic Science. Blood transfusion.

Scientist 105

Scientist Life Science Clinical Research Doctors 105

The Pharma Data

AUGUST 16, 2021

If this genetic material is further replicated, defective RNA copies are produced and the pathogen can no longer spread. Molnupiravir is currently being tested in clinical trials. Based on preliminary clinical trials, the compound promises to be highly effective against Sars-CoV-2. ” Mutations in the genome stop the virus.

RNA 52

RNA Drugs Genome Genomics 52

pharmaphorum

JANUARY 6, 2021

It’s likely no coincidence that Pfizer’s flagship project in 2020 while the design of the logo was being finalised was unquestionably its high-profile alliance with BioNTech on the RNA-based coronavirus vaccine Comirnaty, which provided further evidence of the potential of harnessing genetic toolkits to tackle health issues.

DNA 116

DNA Branding Vaccination Vaccine 116

Olympian Clinical Research

JUNE 2, 2021

One of the most frustrating things about atopic dermatitis is that doctors still don’t know exactly what causes the condition, which means that most treatments are centered around symptom mitigation rather than addressing the root cause. Eczema Clinical Trials In Tampa & Largo. What Causes Atopic Dermatitis?

Clinical Trials 52

Clinical Trials Clinical Trials Clinical Research Trials 52

FDA Law Blog

DECEMBER 7, 2022

We know, for example, the use of viral vectors results in antibody production that could inhibit future administration of gene therapies using the same or similar viruses, which will inform treatment decisions by patients with their doctors. This product was developed by uniQure prior to being licensed to CSL Behring.

Gene Therapy 119

Gene Therapy Gene FDA Approval Production 119

pharmaphorum

FEBRUARY 16, 2022

She received a cord blood transplant to treat the AML, receiving stem cells carrying a genetic mutation (CCR?5/?32) The case study described at the Conference on Retroviruses and Opportunistic Infections (CROI) this week involved a mixed-race woman who had been on antiretroviral therapy (ART) for four years before being diagnosed with AML.

Gene Editing 69

Gene Editing Research Gene Genetics 69

Olympian Clinical Research

NOVEMBER 6, 2023

While the exact cause is unknown, factors like genetics, inflammation, and hormones play a role. Apply topical medication as prescribed by your doctor if needed. Apply a topical or oral anti-inflammatory medication as prescribed by your doctor. You can contribute to this essential work by participating in our clinical trials.

Hormones 52

Hormones Doctors Doctor Clinical Trials 52

The Pharma Data

MAY 9, 2022

Currently, after surgery, doctors examine a specimen of a tumor under a microscope and grade it one, two or three in its aggressiveness. The paper was published today in Nature Genetics. “Eventually we hope to tailor medical therapy to the genetic changes within each individual person’s meningioma,” Magill said.

Drugs 52

Drugs Scientist DNA Genetics 52

XTalks

DECEMBER 20, 2023

The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy. 9. 10 Trends and Statistics for Clinical Trials in 2023 Several key trends in clinical trials have been shaping the landscape of the field this year.

Life Science 59

Life Science Clinical Trials Clinical Trials FDA Approval 59

The Pharma Data

OCTOBER 14, 2020

When the researchers controlled the data for age, education, heart and blood vessel disease, depression and genetic risk of Alzheimer’s disease, they reported that people with severe apathy at the start of the study had 80% higher odds of having dementia later in life. The study findings were published online Oct. 14 in Neurology.

Doctors 52

Doctors Doctor Research Development 52

Drug Discovery World

SEPTEMBER 5, 2022

Tumours (yellow) pervade the lungs of mice genetically prone to small cell lung cancer (left) on this computed tomography scan. Phase I trial on the horizon. We’re talking to a number of other groups about starting a Phase I clinical trial as soon as possible,” Mosammaparast said. This has not changed for 30 years.

Protein 52

Protein Drugs Medicine Research 52

pharmaphorum

OCTOBER 6, 2022

By reducing the disease to its key components, a computer-readable network of disease markers was characterised using genetic, biomarker, and clinical data; the model was also populated with ‘disease vs control’ data. Not only does this make drug discovery more efficient, but it is also less resource intensive. The future of in silico.

In-Vivo 98

In-Vivo Research Genetics Drugs 98

XTalks

SEPTEMBER 22, 2020

“Helping people understand and manage their heart health has always been a priority for Fitbit, and our new ECG app is designed for those users who want to assess themselves in the moment and review the reading later with their doctor,” Eric Friedman, Fitbit co-founder and CTO said in a statement.

Gene 107

Gene Doctors Doctor Clinical Trials 107

Olympian Clinical Research

FEBRUARY 17, 2022

However, genetic abnormalities or other health problems may be involved in the condition. When diagnosing PN, a doctor may perform a skin biopsy to look for signs of the condition, such as thickened skin and inflammation. Through clinical trials, new treatments are developed and made available to those who need them.

Clinical Trials 52

Clinical Trials Clinical Trials Clinical Research Trials 52

XTalks

JUNE 30, 2023

Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII). Hemophilia A affects approximately one in 10,000 individuals and has a higher incidence than hemophilia B. SVB has forecasted around $2.2 billion in peak annual sales of Roctavian.

Gene Therapy 98

Gene Therapy Gene FDA Approval Antibody 98

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52

pharmaphorum

SEPTEMBER 28, 2022

Minor variations in one’s genetic makeup can impact individual responses to medication. Understanding these differences across diverse populations can help doctors and HCPs determine what medication and dosage best fit a patient. ” All of Us is currently looking at ways to share its data with the for-profit sector.

Research 98

Research Medicine Clinical Trials Clinical Trials 98