An experimental gene therapy that’s applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease. Experts called the findings “remarkable,” and said they bring hope of a better quality of life to children and young adults living with the condition, called dystrophic epidermolysis bullosa (DEB). The disease affects about 3 out of every 1 million people. It’s caused by a flawed gene that renders the body unable to produce a particular collagen — a “glue” between the skin layers that is essential to its strength and integrity. Kids born with DEB are sometimes called “butterfly children” because their skin is so fragile, even an ordinary bump or friction can cause blistering that progresses to painful open wounds. In the most severe cases, infants have blisters or missing skin at birth, or soon after. Those children typically develop widespread scarring over their bodies and can have eye inflammation that impairs their vision. Blisters and scarring also arise along the lining of the mouth, throat and digestive tract — which can interfere with eating and cause malnutrition. As young adults, people with DEB face a high risk of squamous cell carcinoma, a form of skin cancer that is normally highly curable, but in a person with DEB often proves deadly. There has never been any specific treatment for DEB.…  read on >  read on >

Having short, painful headaches for many days or even weeks in a row may signal that you’re more likely to have other medical woes, researchers say. These “cluster headaches” are extremely painful and can last from 15 minutes to three hours at a time. And people who have them may be more than three times more likely to have other medical conditions, such as heart disease or mental disorders, the new study found. “Around the world, headaches have an incredibly negative impact on people’s quality of life, both economically and socially,” said study author Caroline Ran, of the Karolinska Institute in Stockholm, Sweden. “Our results show that people with cluster headaches not only have an increased risk of other illnesses, those with at least one additional illness missed four times as many days of work due to sickness and disability than those with just cluster headaches. They also have a higher chance of a long-term absence from work,” Ran explained. The study involved more than 3,200 Swedes, aged 16 to 64, who had cluster headaches. Most were men, because men are more likely to have cluster headaches. The researchers compared that group to 16,200 people without the headaches who were similar in age, gender and other factors. The investigators looked at work records and disability benefits to determine how many days a year people were…  read on >  read on >

An experimental drug may one day be a cure for eczema, a new trial suggests. The drug, rocatinlimab, is a monoclonal antibody that researchers found prevented the recurrence of the symptoms of the skin condition for up to 20 weeks after treatment was stopped. “Patients ask us in the clinic all the time, ‘Can I stop the medication, or can I start doing it much less often?’ Right now we don’t have those medications available,” said lead researcher Dr. Emma Guttman, professor and system chair of dermatology and immunology at the Icahn School of Medicine at Mount Sinai in New York City. “Of course, it’s important in adults, but imagine, in the future, how important this is in children. This drug may cause disease modification because it works on memory T-cells,” she explained. The drug works by blocking OX40 — an immune molecule that activates inflammatory cells that play a role in the development of eczema, also called atopic dermatitis. The drug also prevents memory T-cells from storing the key to eczema. “Because it works on memory T-cell formation, the cells that remember that the patient has the disease, maybe these cells will not come back to cause the disease again,” Guttman said. Atopic dermatitis is a debilitating chronic inflammatory skin disease, which often develops at a very young age, causing the skin to become…  read on >  read on >

Shortages of the ADHD drug Adderall are expected to continue for months, forcing families to scramble for ways to deal with their children’s attention deficit hyperactivity disorder (ADHD). The shortage has caused headaches and hassles for parents like San Diego mom Jackie Meader, who has been “flustered, rushed and out of sorts” since her 16-year-old son’s prescription ran out about a month ago. “He’s telling me he’s having trouble at school, can’t focus. He’s struggling, and it’s not just a matter of not being able to focus on things. He’s having trouble staying awake,” Meader told KGTV News in San Diego. An abrupt halt to Adderall treatment can cause physical symptoms like lethargy and fatigue, but some kids might become jittery, said Dr. Anish Dube, chair of the American Psychiatric Association’s Council on Children, Adolescents and their Families. It also might cause the child to feel hungry and eat more, since stimulants tend to decrease appetite, Dube said. But mainly, kids who are off their Adderall are likely to have their ADHD symptoms return, Dube said. “Particularly if Adderall had been effective in helping with these symptoms, when you have it being stopped, then you might have a return of the original symptoms that the child had,” Dube said. The FDA first declared a shortage of the immediate-release formulation of Adderall on Oct. 12. The…  read on >  read on >

For some people, dozens of U.S.-approved drugs can lead to a rare but often fatal brain infection. Researchers have now confirmed a strong link between four genetic mutations and this illness, called progressive multifocal leukoencephalopathy (PML). A new study found that in people taking PML-inducing drugs, having one of four genetic variants increased the odds of developing PML 8.7 times on average. One of the variants increased risk 33-fold. The findings give doctors the insight to screen out patients with the highest risk for the condition before prescribing these drugs. The U.S. Food and Drug Administration has put its most serious warning — called a Black Box Warning — for PML on eight approved medications. More than 30 drugs carry other PML warnings, and cases have been reported in connection with more than 75 drugs treating everything from multiple sclerosis (MS) to blood cancer, rheumatoid arthritis, Crohn’s disease and organ transplant rejection. This has the potential to affect a lot of people. Nearly 1 million Americans have MS, for example. Another 1.5 million have blood cancers commonly treated with PML-inducing drugs. About 850,000 Americans have received organ transplants, the study authors noted. As more immunosuppressant medications are developed, drug-induced PML is on the rise, with more than 500 cases listed in the FDA’s adverse event reporting system in 2021. “It’s critical to be able to…  read on >  read on >