Bio Pharma Dive

OCTOBER 10, 2024

Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.

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Fierce Pharma

DECEMBER 21, 2023

As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their CRISPR-based gene-editing therapy Casgevy, one doctor who treats sickle cell disease patients is already | As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their world-first CRISPR-based gene therapy Casgevy, one doctor (..)

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pharmaphorum

NOVEMBER 12, 2021

It could also be made more powerful by incorporating personal genetic information and gene expression profiles in tissues such as the lungs, they suggest. The post AI tool may help doctors select best drugs for COVID patients appeared first on.

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Doctors Doctor Drugs Gene Expression 135

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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Gene Therapy Gene Genetics Doctors 98

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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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

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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Gene Therapy Gene Protein Trials 110

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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FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials. One family’s daughter was the first U.S.

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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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Gene Therapy Doctor Doctors Gene 100

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 Doctors Doctor 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 Doctors Doctor 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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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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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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Doctor Doctors Drugs Packaging 98

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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Doctor Doctors 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 Doctors Doctor 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

Trialfacts

MAY 27, 2024

This study will use brain imaging, genetics, questionnaires, and other assessments to map the brain systems and genes that underlie risk and resilience for mental health problems. Click Here to Check Your Eligibility for this Study The post Get Involved in Understanding Your Brain and Genes appeared first on Trialfacts.

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pharmaphorum

SEPTEMBER 21, 2021

Doctors are often unfamiliar with these conditions. Doctors are heavily reliant on the observations of parents or caregivers and are constrained by the ability of the patient to convey symptoms they are experiencing. To complicate matters further, CNS conditions are often very difficult to identify, characterise and treat.

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Pharmaceutical Technology

AUGUST 12, 2022

We have patients calling us saying, ‘I want to get into this site, but they say [it will be] three months before they can see me,’ and it's not because the doctors don't want to do it. Gene therapies, another area highlighted in the strategy, are also being explored in the ALS clinical landscape, says Cudkowicz.

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Research Gene Therapy Drugs Trials 246

Trialfacts

MAY 27, 2024

Study Location Research Centre: Monash Biomedical Imaging Location: 770 Blackburn Road, Clayton 3168 Lead Doctor: Professor Alex Fornito Ethics Committee: This study has been reviewed and approved by the Monash University Human Research Ethics Committee (Project #12692) Background Attention, young men! Contents Background Why Participate?

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Gene Hormones Research Doctor 52

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

World of DTC Marketing

JANUARY 31, 2021

Ex vivo (where cells are genetically modified outside the body) cell and gene therapies have generated considerable excitement on their potential to cure previously incurable diseases. Then there are ever increasing health insurance and hospital costs at a time when less people are going to the doctor’s office because of COVID-19.

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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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XTalks

NOVEMBER 18, 2021

Through genetic testing, it was revealed that Juanea carries the gene for the heart condition. Juanea said she felt “really really bad” when she found out she was a carrier of a mutated gene involved in HCM, but then had to come to “grips” with the fact that “this was something beyond [her] control.”.

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Doctors Doctor Genetics Gene 98

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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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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