STAT News

JANUARY 24, 2023

Then, months later, doctors delivered gene therapy directly to her brain. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep. Read the rest…

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pharmaphorum

SEPTEMBER 13, 2021

AbbVie has expanded its ophthalmology pipeline via a licensing deal with Regenxbio, paying $370 million upfront to get its hands on a gene therapy for wet age-related macular degeneration (AMD) and other eye diseases. The post AbbVie spies potential in Regenxbio eye disease gene therapy appeared first on.

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Gene Therapy Gene Sales Trials 97

FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Gene Therapy Gene FDA Approval Production 119

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023. The post Sarepta says early filing for DMD gene therapy is back on appeared first on.

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Gene Therapy Gene Genetics Trials 105

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. 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).

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Gene Therapy Gene FDA Approval Antibody 98

Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. Continuing advancements within this field deliver new hope to doctors and patients, transforming disease outcomes for previously incurable indications. Further opportunities arise from the industry’s move towards sustainability.

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Drugs Gene Therapy Production Manufacturing 295

BioSpace

NOVEMBER 16, 2021

After 32 years, gene therapy pioneer Guangping Gao, Ph.D., is finally seeing the fruits of the doctoral thesis research he started in 1989.

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Scienmag

MARCH 24, 2021

to UD lab will build on breakthrough discovery of gene linked to eye disorders Credit: Photo by Evan Krape When University of Delaware doctoral student Sandeep Aryal took the initiative to learn additional research techniques to study proteins present in the eye, his work resulted in the discovery of a new gene, known as […].

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Gene Doctor Doctors Protein 94

pharmaphorum

NOVEMBER 3, 2020

Ring Chromosome 20 Syndrome, or (R)20, is an ultra-rare form of epilepsy with a devastating impact – yet despite huge leaps forward in gene sequencing in recent years, diagnoses are going down instead of up. There are no clinical practice guidelines, meaning doctors treat on a case-by-case basis. appeared first on.

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Gene Sequencing Gene Genome Genomics 118

Scienmag

JUNE 29, 2021

Scientists and doctors at University College London Great Ormond Street Institute of Child Health (UCL GOS ICH) and Great Ormond Street Hospital (GOSH) have given hope of a gene therapy cure to children with a rare degenerative brain disorder called Dopamine Transporter Deficiency Syndrome (DTDS).

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Gene Therapy Gene Doctor Doctors 58

Medical Xpress

MAY 16, 2023

That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. They study gene variation and mutations that cause rare genetic diseases. Sometimes scientists arrive at new findings and discoveries by chance.

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XTalks

NOVEMBER 15, 2024

You know how to protect against COVID and flu so ask your pharmacist or doctor about scheduling an RSV vaccine, too.” The commercial talks about RSV being a “highly contagious virus” and that people 60 years of age and older have a higher risk of being hospitalized from RSV, making vaccination against the virus important.

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Vaccination Vaccine Gene Therapy RNA 105

XTalks

JANUARY 21, 2025

A doctor inserts the catheter through a small incision in a vein in the groin and guides it to the heart. The catheters tip is positioned at various locations within the heart, where the doctor uses a generator to deliver RF energy. Companion diagnostic for Pfizers hemophilia B gene therapy Beqvez (idanacogene elaparvovec-dzkt).

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FDA Approval Manufacturing Development RNA 105

STAT News

APRIL 3, 2023

They had been modified in a lab by adding a functional gene to compensate for a defective one. Conner’s doctors expect that this groundbreaking gene therapy, which costs a staggering $3 million a patient, will stave off a fatal degenerative brain disease and save his life.

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Gene Therapy Nurses Gene Doctors 98

STAT News

APRIL 6, 2023

… A major obstacle looms for the drugs of the future – not enough doctors know how to administer them , The Wall Street Journal writes. Promising therapies for some diseases are administered through lumbar punctures, which are not a routine part of a doctor’s daily practice.

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Scienmag

JUNE 9, 2021

First study to show gene expression changes in divers with ‘the bends’ reveals key role of genes for inflammation and immunity For over a century, researchers have known about “the bends”, a serious condition affecting scuba divers. Doctors do not yet have a definitive test […].

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Gene Expression Gene Doctors Doctor 75

pharmaphorum

JANUARY 26, 2021

Under the leadership of CEO Vas Narasimhan, who took over in 2018, the company has focused on using digital technology to engage with both patients and doctors. Marie-France Tschudin, president of Novartis’s pharmaceuticals business, said digital technology had helped to improve productivity and its interactions with doctors.

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Doctors Doctor Sales Clinical Trials 111

pharmaphorum

DECEMBER 2, 2021

Whether catching up with relatives, attending a business meeting, or even visiting a doctor, it became commonplace to conduct this type of activity via a screen. According a Pew Research study, 81% of participants said they have used video calls to talk to others since the onset of the pandemic. Tapping into data.

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Genetics Doctors Doctor Pharmacy 145

Medical Xpress

DECEMBER 2, 2022

Changes in a single gene open the door for harmful gut bacteria to set off the inflammation that drives Crohn's disease, according to a new study led by Weill Cornell Medicine and NewYork-Presbyterian investigators. These findings could one day help doctors better select targeted treatments for patients with this immune disorder.

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Bacteria Genetics Doctor Doctors 75

Camargo

JULY 30, 2021

In 1981, I got my doctorate degree in Clinical Pharmacology, but more importantly, my research was heavily involved in neonatal medicine, developing drugs there. That has to do a lot with the work in gene therapy and rare disease and the ultra-rare space because there are 7,000 rare diseases that have been identified.

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Clinical Research Research Gene Therapy Drugs 226

pharmaphorum

OCTOBER 21, 2021

Many are still predicting multibillion-dollar sales for the drug, albeit with a longer runway, despite reservations about its clinical efficacy among doctors and payers. Analysts meanwhile have suggested that Aduhelm may start to pick up momentum if that review is positive.

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Sales Doctors Doctor Gene Therapy 122

Pharmaceutical Technology

FEBRUARY 23, 2023

It covers a broad range of techniques from the less invasive practice of intermittent fasting to drastic and invasive procedures, such as the implanting of microchips or injecting of gene-editing enzymes. nurses, doctors, and hospital auxiliary staff) to meet these needs.

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Gene Editing Regulation Production Nurses 278

pharmaphorum

OCTOBER 27, 2020

Novartis’ pharmaceutical sales recovered in the third quarter of this year after an earlier COVID-19 decline, as new drugs like gene therapy Zolgensma gathered pace. . Correcting for currency fluctuations, net sales were flat at $12.3

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pharmaphorum

SEPTEMBER 12, 2022

Across LabCorp , we innovate through science and technology, with access to approximately 2,500 [doctors] and PhDs and over 700 patients. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.

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Genome Genomics Clinical Trials Clinical Trials 105

Pharmaceutical Technology

JULY 28, 2022

Roche’s Susvimo (ranibizumab sustained release formulation) follows a similar theme; patients who are implanted with Susvimo are expected to visit their doctor only twice a year to have the implant refilled so that the medicine can be delivered to their eyes continuously. The therapy received FDA approval in October last year.

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Marketing Gene Therapy FDA Approval Development 130

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.

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Gene Doctors Doctor Clinical Trials 107

WCG Clinical

JUNE 4, 2024

I am in the Doctorate in Nursing Practice program at the University of California, San Francisco, and I’m really looking forward to, becoming the first person in my family on either side to achieve a doctorate degree. We started experimenting with different gene therapies and antisense therapies.

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Nurses Gene Therapy Doctors Doctor 52

The Pharma Data

DECEMBER 17, 2020

Doctor Johnson has had an inspiring career that encompasses several aspects of the pharmacy industry. In keeping with her pioneering spirit, she recently expanded into Pharmacogenomics (PGx) gene testing for patients. Doctor Johnson aspires to incorporate the latest technologies available in healthcare to care for her patients.

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Medicine Pharmacy Doctors Doctor 52

XTalks

APRIL 6, 2022

Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. 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. Live and On-Demand: Thursday, April 21, 2022, at 11am EDT (4pm BST/UK).

DNA 105

DNA In-Vitro Doctors Doctor 105

pharmaphorum

MAY 6, 2021

One example that falls into this category was Game Doctor’s Remedy Quest, launched into schools last year with the help of a £50,000 grant from the UK government, which is designed to educate young people about COVID-19 by simulating virus prevention and control scenarios. .

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Genetic Disease Protein RNA Doctor 104

DM Clinical Research

DECEMBER 15, 2020

However, they do usually mean you should see a doctor. Patients with infection symptoms should see their doctor immediately. Periodic fever syndromes are usually caused by gene mutations. If you suffer from fevers with no known cause, then ask your doctor about these conditions. Talking to a Doctor.

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Doctor Doctors Clinical Research Gene 45

pharmaphorum

JANUARY 29, 2021

They studied 56 medicines, including antidepressants, antibiotics, stomach ulcer treatments and painkillers where there are known drug-gene interactions. And they found that in more than one in five occasions (21.1%) there was the potential for a doctor or pharmacist to take action under international pharmacogenetic guidelines.

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Genetics Pharmacy Medicine Doctor 98

XTalks

JANUARY 17, 2024

There are many aspects of oncology drug development that are driven by the unique nature of the treatments being developed and the needs of the patients, their caregivers and the doctors that treat them. Morrison remarked that the latest ESMO was unique in its broad discussion scope, touching on various innovative treatments.

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Clinical Trials Clinical Trials Trials Gene Therapy 124

pharmaphorum

FEBRUARY 28, 2022

Many of the small challenges that face individual doctors and patients in seeking to find a solution for a rare disease also contribute to the difficulty of researching and developing an effective treatment. Once diagnosed, only 5% of rare diseases actually have an effective treatment. The outlook for rare disease R&D. Work to be done.

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Gene Therapy Development Gene Medicine 104

DM Clinical Research

DECEMBER 15, 2020

However, they do usually mean you should see a doctor. Patients with infection symptoms should see their doctor immediately. Periodic fever syndromes are usually caused by gene mutations. If you suffer from fevers with no known cause, then ask your doctor about these conditions. Talking to a Doctor.

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Doctor Doctors Gene Clinical Research 40

pharmaphorum

JUNE 24, 2022

FoCus investigator Prof Karl Heinz Weiss of Salem Medical Centre Heidelberg said the results “have the potential to reframe the way doctors can think about the disease given that current therapies focus on removing copper from the blood.”

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