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Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement Tuesday. The patient died after experiencing acute liver failure. Testing showed he also had a recent cytomegalovirus infection, which was a possible contributing factor, according to the treating physician.
“Although it is not a new safety signal and the benefit-risk of Elevidys remains positive, acute liver failure leading to death represents a severity of acute liver injury not previously reported for Elevidys, which to date has been used to treat more than 800 patients in clinical trials or as a prescribed therapy,” Sarepta said in the statement.
Acute liver injury is a possible side effect of delandistrogene moxeparvovec and other adeno-associated virus (AAV)-mediated gene therapies and is highlighted in the treatment’s prescribing information.
Banning Direct-to-Consumer Drug Ads Won’t Work
As a reformed hypochondriac, I’m extremely vulnerable to the allure of pharmaceutical ads. I self-referred for GLP-1s years ago after seeing advertisements, and even though I am a diabetic, what followed were horrific health outcomes: hospitalizations, extensive procedures, scans, MRIs, pancreatitis, anemia, and thousands in medical expenses.
So I understand why new Health and Human Services Secretary Robert F. Kennedy Jr. has long called for a ban on pharmaceutical advertisements on TV.
But that seems unlikely to happen. The pharmaceutical industry is simply too strong — it spends billions of dollars on direct-to-consumer advertising and lobbying efforts.
Fewer California Kindergarten Students Immunized Against Measles Last Year, New Data Show
The Los Angeles Times reported:
California reported a decline last year in the share of kindergarten students who were immunized against measles. Sixteen counties reported that measles vaccination rates fell below herd immunity against one of the most contagious diseases. California’s childhood vaccination rates are still higher than in the U.S. overall. But experts worry that deep political discord, along with widespread disinformation online, will only make it harder to reverse the downward trend.
Despite having some of the nation’s strictest school vaccination laws, California reported a decline last year in the share of kindergarten students who were immunized against measles, including in 16 counties where students no longer have herd immunity against one of the most contagious diseases.
New data from the California Department of Public Health show that last year, 96.2% of California students in transitional kindergarten and kindergarten were vaccinated against measles, mumps and rubella in the 2023-24 school year, down from 96.5% the year before. And 93.7% of kindergarten students were up to date on all their immunizations, down from 94.1% in the same period the previous year. Data on vaccination rates for first-grade students, which are usually higher, were not yet available.
‘It’s Not a Vaccine, It’s a Shot’: Uncovering a New Trend in Vaccine Skepticism
It has long been recognized that attitudes towards vaccines may be vaccine-specific, so that people may take up some, but not others. On July 26, 2021, the following statement was posted on Twitter (later renamed X) about the COVID-19 vaccine: “It’s not even a real vaccine. You can catch COVID-19 and also spread it if you are vaccinated. You don’t catch polio or MMR after you are vaccinated.”
My colleagues and I came across this comment and many liked it while analyzing a nine-million-word dataset consisting of tweets about the COVID-19 and MMR vaccines posted between 2008 and 2022, to learn more about vaccine skepticism.
We discovered that the author of this tweet is not alone in questioning the status of the COVID-19 vaccines as vaccines, and comparing it to others.
The FDA Finally Visited an Indian Drug Factory Linked to U.S. Deaths. It Found Problems.
The U.S. Food and Drug Administration (FDA) has found problems at an Indian factory that makes generic drugs for American patients, including one medication that was manufactured there and has been linked to at least eight deaths, federal records show.
The agency inspected the factory after a ProPublica investigation in December found that the plant, operated by Glenmark Pharmaceuticals, was responsible for an outsized share of recalls for pills that didn’t dissolve properly and could harm people.
Among the string of recalls, the FDA had determined last year that more than 50 million potassium chloride extended-release capsules had the potential to kill U.S. patients. Still, ProPublica found, the FDA had not sent inspectors to the factory in Madhya Pradesh, India, since before the COVID-19 pandemic.
WHO Prequalifies First Maternal Respiratory Syncytial Virus Vaccine
The World Health Organization reported:
On March 12, 2025, the World Health Organization prequalified the first maternal respiratory syncytial virus (RSV) vaccine to protect infants against one of the most common causes of acute lower respiratory infections in children globally. Each year, RSV causes more than 3.6 million hospitalizations and about 100,000 deaths in children under five years of age. About half of these deaths occur in infants younger than six months of age.
The majority of pediatric RSV deaths occur in low- and middle-income countries where there is limited access to supportive medical care. Currently, there are no specific treatments for RSV infection, making supportive measures the main line of therapy. Preventive measures, such as vaccines, are key to reducing cases of pneumonia and bronchiolitis, decreasing hospitalizations and oxygen use, and saving infant lives globally.
After decades of research, there are currently two licensed immunization products for prevention of RSV disease in young infants: the maternal vaccine given to pregnant women in the third trimester to protect their babies and a long-acting monoclonal antibody administered to infants from birth just before or during the RSV season.
Purdue Pharma Submits Opioid Lawsuit Settlement Plan, Including up to $7 Billion Cash From Sacklers
Purdue Pharma asked a bankruptcy judge late Tuesday to consider the latest version of its plan to settle thousands of lawsuits over the toll of the powerful prescription painkiller OxyContin, a deal that would have members of the Sackler family who own the company pay up to $7 billion. The filing is a milestone in a tumultuous legal saga that has gone on for more than five years.
Under the deal the family members — estimated in documents from 2020 and 2021 to be worth about $11 billion — would give up ownership of the company in addition to contributing money over 15 years with the biggest payment up front.
Family members resigned from Purdue’s board, stopped receiving money from the company and ceased other involvement before it filed for bankruptcy protection in 2019 as it faced lawsuits from thousands of state and local governments, plus others.
Single Vaccine Dose Shows 84% Effectiveness Against Mpox, but Insufficient for HIV Patients
Universitätsmedizin Berlin has found that a single dose of the Imvanex vaccine provides protection against mpox with 84% effectiveness. For people with HIV, however, a single dose of the vaccine fails to offer sufficient protection. All at-risk groups, and people with HIV in particular, should therefore receive the second dose of the vaccine as recommended. The results of the study have now been published in The Lancet Infectious Diseases.
Since a wave of infections with the mpox virus swept around the world in 2022, Germany’s Standing Commission on Vaccination has recommended that people at an elevated risk of mpox infection receive an Imvanex vaccination. Originally developed to protect against smallpox, the European Medicines Agency (EMA) approved Imvanex for use to protect against mpox in July 2022 in response to the public health emergency.
The mpox virus is related to the original smallpox virus. The EMA’s decision to approve Imvanex was based on laboratory data showing that the vaccine offers cross-protection. Until now, however, the extent of this protective effect was not clear, especially for at-risk groups.
