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A young boy died in a trial for Pfizer’s experimental gene therapy for Duchenne muscular dystrophy, the company told patient advocates Tuesday. The boy was enrolled in Daylight, a trial studying the treatment in boys aged 2 or 3. The boy had received the therapy early last year, Pfizer told the advocates in a note posted online by Parent Project Muscular Dystrophy. Pfizer said it had not yet determined precisely what happened or whether it was linked to the treatment. Deaths and severe side effects linked to gene therapy have generally happened soon after dosing.

For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy. On Thursday evening, the FDA announced it expanded the approval of Elevidys, Sarepta’s Duchenne gene therapy, to cover nearly all patients, regardless of age or wheelchair status, despite the fact that the drug failed a large, Phase 3 trial last year. That decision, documents concurrently released by the agency show, was made almost exclusively by Peter Marks, the agency’s director of the Center for Biologics Evaluation and Research. He...

I remember being a kid and seeing my grandmother without her dentures for the first time. It was a harrowing experience. Now my dad has dentures so, genetically speaking, I’m several decades out from needing some myself. However, it’s possible that modern medicine will solve the issue of lost teeth by then, thanks to a new drug that's about to enter human trials. The medicine quite literally regrows teeth and was developed by a team of Japanese researchers, as reported by New Atlas. The research has been led by Katsu Takahashi, head of dentistry and oral surgery at Kitano Hospital....

The world's first human trial of a drug that can regenerate teeth will begin in a few months, less than a year on from news of its success in animals. This paves the way for the medicine to be commercially available as early as 2030. The trial, which will take place at Kyoto University Hospital from September to August 2025, will treat 30 males aged 30-64 who are missing at least one molar. The intravenous treatment will be tested for its efficacy on human dentition, after it successfully grew new teeth in ferret and mouse models with no significant side...

The world's first human trial of a drug that can regenerate teeth will begin in a few months, less than a year on from news of its success in animals. This paves the way for the medicine to be commercially available as early as 2030. The trial, which will take place at Kyoto University Hospital from September to August 2025, will treat 30 males aged 30-64 who are missing at least one molar. The intravenous treatment will be tested for its efficacy on human dentition, after it successfully grew new teeth in ferret and mouse models with no significant side...

Researchers in Japan are currently working on a medication that would allow people to grow a new set of teeth, with a clinical trial slated for July 2024.

thousands of elderly COVID patients in the United Kingdom were secretly euthanized in April 2020 ... This disturbing claim came from an investigation directed by Wilson Sy, director, Investment Analytics Research Australia, and ... The alleged euthanasia claim seems unlikely because in the U.K., it is regarded as either manslaughter or murder by the National Health Service ... Having had a career in analytics, I was skeptical. I reviewed the ResearchGate investigation documentation fully expecting to find fake news. Instead, I found that the report was exceptionally well researched and documented, and the claim appears valid. The truth was buried...

Pfizer has stunned the medical world, completing the $43 billion acquisition of Seagen, a small drug company that treats turbo cancers and barely makes $2 billion per year. The acquisition means Pfizer becomes the largest oncology company in the world, capable of treating most turbo cancers caused by mRNA vaccines. However, the nature of the acquisition has left many people scratching their heads. Why would Pfizer, flush with the enormous profits it has reaped through its mRNA vaccine, overpay $43 billion for a small cancer drug company? Pfizer does not need the cash. It will also issue $31 billion in...

Now that the pandemic has ended, researchers are urging regulatory agencies to consider the safety issues associated with the rapid approval of COVID-19 vaccines—and to correctly classify messenger RNA (mRNA) vaccines as gene therapy products (GTPs) to prevent pharmaceutical companies from bypassing regulatory standards. According to a paper published in the International Journal of Molecular Sciences on June 22, COVID-19 mRNA vaccines, by mode and action, are gene therapy products and should adhere to different regulatory standards. Yet U.S. and European regulatory agencies have not classified COVID-19 mRNA vaccines as gene therapy products, which has allowed them to be regulated...

The FDA-approved lung cancer drug osimertinib (sold under the brand name Tagrisso) slashes the risk of death by 51% for certain patients whose cancer is caught early, according to new trial data. “This should be the new standard of care for these patients,” Nathan Pennell, co-director of the Cleveland Clinic Lung Cancer Program, who wasn’t involved in the drug’s development, said in a statement for the American Society of Clinical Oncology. The drug: Lung cancer is the second-most common cancer in the world, with 2.2 million new diagnoses every year. The vast majority fall into a category called “non-small cell...

Researchers found [a] test can predict whether a bowel cancer patient will benefit from chemotherapy. It is thought that this could spare patients who will not benefit from treatment from unnecessary toxicity and debilitating side effects. The genetic test is already used as part of standard of care in the UK and worldwide to predict patients' responses to other targeted cancer drugs, meaning doctors could apply it to guide chemotherapy straight away. In bowel cancer, responses to last-line chemotherapy treatment trifluridine/tipiracil vary greatly between patients—with some patients showing good, long-term responses, and others seeing no benefits. The researchers found that...

ntil recently, CRISPR—the gene-editing technology that won scientists Jennifer Doudna and Emmanuelle Charpentier the 2020 Nobel Prize in chemistry—sounded more like science fiction than medicine; lab-created molecular scissors are used to snip out problematic DNA sections in a patient’s cells to cure them of disease. But soon we could see regulators approve the very first treatment using this gene-editing technology in an effort to combat rare inherited blood disorders that affect millions across the globe. In a $900 million collaboration, rare disease specialist Vertex and CRISPR Therapeutics developed the therapy, dubbed exa-cel (short for exagamglogene autotemcel). It has already amassed...

Numbers showing the majority of Alberta's COVID-19 hospital admissions are in vaccinated individuals don't tell the entire story, regardless of what some conspiracy theorists believe. Albertans with at least one shot took over more hospital beds than those without this week. But the raw numbers are incomplete, and a dive into the data shows being vaccinated may be more important now than ever before. As of Thursday, 217 unvaccinated Albertans were in hospital with COVID-19, compared to 282 patients with at least one shot. But almost 90 per cent of the province has one dose, which means the "rate per...

Georgian tennis star Nikoloz Basilashvili was forced to drop out of the Sydney Cup due to breathing difficulties and needing treatment from a doctor during his singles match against Greece’s Stefanos Tsitsipas. The 29-year-old and World’s no. 22 tennis player had to end his ATP Cup match early due to breathing difficulties on Wednesday. A doctor and physiotherapist attended Basilashvili while sitting with his team.

At this year’s World Health Summit Stefan Oelrich, President of Bayer’s Pharmaceuticals Division, gave a truly eye-opening speech revealing how the so-called “experts” think and quite frankly, taking his speech in the round, it’s chilling.Every October, the World Health Summit draws international “experts” from academia, politics, the private sector, and civil society to Berlin. The 13th World Health Summit was held in Berlin over the 3 days 24 – 26 October 2021. There were 67 sessions with 377 speakers involving 6,000 participants from 120 nations. The Summit’s partners include: Sanofi, Johnson & Johnson, Pfizer, Roche, Wellcome, Biogen, Novartis, Bayer and...

New laws in Maryland and Montana are the first in the nation to restrict law enforcement’s use of genetic genealogy, the DNA matching technique that in 2018 identified the Golden State Killer, in an effort to ensure the genetic privacy of the accused and their relatives. Beginning on Oct. 1, investigators working on Maryland cases will need a judge’s signoff before using the method, in which a “profile” of thousands of DNA markers from a crime scene is uploaded to genealogy websites to find relatives of the culprit. The new law, sponsored by Democratic lawmakers, also dictates that the technique...

Conceptual illustration of breast cancer cells. (MedicalRF.com/Getty Images) Researchers in Australia have discovered a gene responsible for a particularly aggressive type of hormone-sensitive breast cancer which has tragically low survival rates. "Hopefully this will dramatically improve the poor outcomes these patients currently suffer," said Harry Perkins Institute of Medical Research epigeneticist Pilar Blancafort. It's hard to overstate just how different cancers can be from one another. Even under the umbrella of 'breast cancer' lie several types, such as hormone receptor sensitive, HER2 positive, or non-hormone sensitive breast cancer; within those groups, there are even more types that can respond to...

Gene Simmons is done with the Golden State. The Kiss rocker has announced his departure from California after more than 35 years. “We are moving out of L.A. for a number of reasons: one of which are the tour buses,” Simmons explained to the New York Post. “After a certain point, we have had enough of that, even though we appreciate the attention.” It’s not just the tourists that have gotten to Simmons. The rocker listed “earthquakes, fires and pandemics every year” among his further reasons for moving. Primis Player Placeholder Instead, the Kiss bassist will now reside in a...

A single test can identify the multitude of known sources of genetic variation underlying mismatch repair deficiency syndrome in inherited colon, endometrial, and other cancers, researchers report in The Journal of Molecular Diagnostics. ============================================================ Researchers have developed a new integrated genetic/epigenetic DNA-sequencing protocol known as MultiMMR that can identify the presence and cause of mismatch repair (MMR) deficiency in a single test from a small sample of DNA in colon, endometrial and other cancers. This alternative to complex, multi-step testing workflows can also determine causes of MMR deficiency often missed by current clinical tests. Their results are presented in The...

Damage to the retina is the leading cause of blindness in humans, affecting millions of people around the world. Unfortunately, the retina is one of the few tissues we humans can't grow back. Unlike us, other animals such as zebrafish are able to regenerate this tissue that's so crucial to our power of sight. We share 70 percent of our genes with these tiny little zebrafish, and scientists have just discovered some of the shared genes include the ones that grant zebrafish the ability to grow back their retinas. "Regeneration seems to be the default status, and the loss of...