World of DTC Marketing

JANUARY 1, 2022

1ne: The value proposition of prescription drugs still outweighs potential side effects – Wegovy’s demand quickly exceeded supply after the FDA approved once-weekly semaglutide injections. People don’t want to lose weight through diet and exercise if they can lose it through a prescription drug.

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Marketing Doctor Doctors FDA Approval 244

XTalks

MAY 17, 2024

Xtalks spoke with Richard Philipson, MD, Chief Medical Officer (CMO) at Calliditas Therapeutics, a Sweden-based company with full commercial operations in the US focused on developing treatments for rare diseases to learn more about the continuing evolution of rare disease drug development.

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Development Drugs Clinical Trials Clinical Trials 52

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. The post Data backs AZ’s Wilson disease drug, but with a question mark appeared first on.

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Drugs Gene Therapy Compliance Trials 100

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.

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Development Drugs Trials Medicine 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

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.

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Vaccination Vaccine Drugs Clinical Trials 98

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. At full stretch, researchers are developing various vaccines and drugs – with different degrees of success. Molnupiravir, another antiviral drug candidate, was originally developed to treat influenza.

RNA 52

RNA Drugs Genome Genomics 52

The Pharma Data

MAY 9, 2022

No drugs are approved for these aggressive tumors, which occur in up to 20% of cases and can lead to patient disability or even death. The drug is a newer cancer treatment called abemaciclib. Currently, after surgery, doctors examine a specimen of a tumor under a microscope and grade it one, two or three in its aggressiveness.

Drugs 52

Drugs Scientist DNA Genetics 52

The Pharma Data

DECEMBER 28, 2020

The Corps is a group of doctors, public health professionals and community advocates working to reverse the COVID-19 outbreak and protect our city. About Fulgent Genetics. TEMPLE CITY, Calif., It ensures that anyone with the virus receives care and can safely isolate to prevent the spread. For more information, visit [link].

Genetics 52

Genetics Sales Gene Sequencing Marketing 52

pharmaphorum

DECEMBER 21, 2021

However, despite the work still left to be done, there are now a growing number of treatments in the pipeline and 2020 proved to be a remarkable year for orphan drug approvals, with the US FDA approving more orphan designated treatments than non-orphan drugs. Headway being made.

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Development Genome Genomics Drugs 98

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.

Gene Therapy 87

Gene Therapy Gene FDA Approval Dermatology 87

The Pharma Data

SEPTEMBER 1, 2021

Louis have genetically engineered cells that, when implanted in mice, will deliver a biologic drug in response to inflammation. The approach allows those cells to remain in the body for a long time and secrete a drug whenever there is a flare of inflammation.” million adults in the United States.

Drugs 52

Drugs Engineer Drug Delivery Gene 52

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The disease is also difficult to diagnose and often takes around six years before doctors correctly identify it.

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FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

AUGUST 11, 2022

More than 60 percent of all American adults take prescription drugs , amounting to approximately 131 million individuals. These drugs have been rigorously tested by regulatory bodies around the world before they’re made available to ensure they work as labelled, but despite that, adverse events crop up.

Genome 98

Genome Genomics Drugs Genetics 98

pharmaphorum

AUGUST 13, 2020

Exon-skipping drugs are used to patch the mutations and allow the gene to produce partially functional dystrophin. Viltepso can be used to treat around 8% of DMD patients and previously approved DMD drugs has not been shown to change the course of the disease. In the study, dystrophin levels increased, on average, from 0.6%

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FDA Approval Drugs Gene Trials 52

XTalks

JULY 24, 2024

AI-driven models also help in identifying the most effective treatment regimens based on patient-specific factors, including genetic makeup and treatment history. QDX, a computational drug discovery company, exemplifies this trend. This enables early detection and personalized treatment planning. Nanostics Inc.

Big Data 52

Big Data Clinical Trials Clinical Trials Life Science 52

pharmaphorum

OCTOBER 25, 2021

The company is best known for its saliva-based health and ancestry DNA testing targeted at consumers, but also uses its genetic database to look for new targets for drug development. The transaction is expected to close before the end of the year.

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DNA Genetics Nurses Pharmacy 111

pharmaphorum

APRIL 8, 2021

COVID-19 has been a trial-by-fire showing just how quickly drugs and vaccines can be developed under the right circumstances – but there is much to do to ensure this progress is not lost, said speakers at WIRED Health 2021, including the CEOs of BioNTech and BenevolentAI. . We published our hypothesis in the Lancet on February 4 in 2020.

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Development Drugs Vaccination Vaccine 93

XTalks

NOVEMBER 18, 2021

“The first time I heard the term hypertrophic cardiomyopathy, I was in the doctor’s office in the room getting diagnosed. HCM is a genetic heart condition in which the muscle of the heart thickens, causing it to work harder to pump blood. Through genetic testing, it was revealed that Juanea carries the gene for the heart condition.

Doctor 95

Doctor Doctors Genetics Gene 95

FDA Law Blog

MARCH 13, 2023

Bauer, Senior Regulatory Drug Expert & James E. Valentine — Incorporating patient and caregiver experiences into every phase of drug development has become increasingly prioritized during both development and review ( see, e.g. , previous coverage here ). By Larry J. There have been challenges in gene therapy development.

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

STAT News

APRIL 3, 2023

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. Millions of stem cells that were collected from 6-year-old Conner Hess’s blood in January flowed through the IV and entered his bloodstream.

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Gene Therapy Nurses Gene Doctor 98

The Pharma Data

DECEMBER 17, 2020

7, 2020 — After starting a drug that’s officially approved to treat a type of blood cancer, a young man with type 1 diabetes was able to stop using insulin. 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.

Insulin 52

Insulin Genetics Doctor Doctors 52

pharmaphorum

FEBRUARY 25, 2021

Chaired by the Genetic Alliance, the virtual meeting saw representatives from Scotland, England, Wales, and Northern Ireland discuss the implementation of the UK Government’s Rare Disease Framework. We now know that 80% of rare diseases have a genetic origin. Genomic technology, therefore, has a key role to play in our work,” he said.

Genome 111

Genome Genomics Genetics Medicine 111

pharmaphorum

SEPTEMBER 21, 2021

“We need to ensure qualified patients are diagnosed, identified and engaged early on in drug development”. 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.

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Gene Therapy Gene Clinical Trials Clinical Trials 90

pharmaphorum

JANUARY 15, 2023

Neurological conditions like Multiple Sclerosis (MS) are an incredibly important area of research when it comes to drug discovery, but under that (sometimes mistaken) umbrella diagnosis occur other, rarer diseases, such as Neuromyelitis Optica Spectrum Disorder (NMOSD). Until recently, NMOSD was regarded as an optic-spinal form of MS.

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Antibody Genetics Medicine Drugs 105

pharmaphorum

OCTOBER 6, 2022

These include exposure to various chemicals associated with the war, from chemical warfare agents to pyridostigmine bromide – a preventative drug given to soldiers at risk of exposure to toxic chemicals. Not only does this make drug discovery more efficient, but it is also less resource intensive. Tinker, tailor, soldier, therapy.

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In-Vivo Research Genetics Drugs 98

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

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Genetic Disease Protein RNA Doctors 104

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 Doctor Doctors 111

pharmaphorum

AUGUST 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans. The three drugs required intravenous dosing once a week and work only in around a third of DMD patients.

Gene Therapy 105

Gene Therapy Gene Genetics Trials 105

XTalks

APRIL 6, 2022

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. We’re hoping that this technology could help doctors decide when additional rounds of treatment are needed, and could save lives.”.

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DNA In-Vitro Doctor Doctors 103

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.

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DNA Branding Vaccination Vaccine 116

XTalks

JUNE 30, 2023

The US Food and Drug Administration (FDA) approved BioMarin’s Roctavian, an adeno-associated virus (AAV) vector-based gene therapy, for the treatment of adults with severe hemophilia A. 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

XTalks

MAY 20, 2022

They also assist in the testing of medical devices and development of drug products. 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. Genetic Science. What is it Like to Work as a Clinical Scientist? Microbiology.

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Scientist Life Science Clinical Research Doctor 104

pharmaphorum

FEBRUARY 16, 2022

She received a cord blood transplant to treat the AML, receiving stem cells carrying a genetic mutation (CCR?5/?32) Having a third functional cure of HIV nevertheless does give pointers to how a more widely applicable cure might be developed in the future, for example using gene-editing drugs to introduce CCR5?32

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Gene Editing Research Gene Genetics 69

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. The result of this approach has seen a number of breakthroughs in the development of orphan drugs. A growing pipeline.

Gene Therapy 104

Gene Therapy Gene Development Medicine 104

XTalks

DECEMBER 20, 2023

These advancements and trends addressed important industry challenges and offered opportunities to foster innovations and solutions in areas including drug development, healthcare and medical devices. The shortages have been partly blamed on the off-label prescribing of the drugs for weight loss.

Life Science 59

Life Science Clinical Trials Clinical Trials FDA Approval 59

FDA Law Blog

DECEMBER 7, 2022

Of course, neither “cure” nor permanency are the bar for FDA to find that a new drug is effective; simply because there is the potential for something to be a “cure” or offer permanent/near-permanent effects does not mean it should be required to be such. This product was developed by uniQure prior to being licensed to CSL Behring.

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Gene Therapy Gene FDA Approval Production 119