Second Patient Death Reported With Gene Therapy for Muscular Dystrophy
Sarepta reported the death over the weekend and provided additional details about its response, which includes pausing shipments of the therapy Elevidys for older patients who are no longer able to walk. The one-time treatment is approved for children with a genetic variant of Duchenne's muscular dystrophy, which causes weakness, loss of mobility, and early death in males. Elevidys is the first gene therapy approved in the US for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. The second death, like an earlier one reported in March, occurred in a teenage boy who suffered a fatal case of acute liver injury, a known side effect of the therapy. Older patients receive a larger dose of the therapy.
Sarepta said it would pause a study in those patients and assemble an expert panel to recommend new safety protocols for taking the drug. Those changes are expected to include increased use of immune-system-suppressing drugs, company executives said Monday. The liver injury associated with the therapy is thought to be connected to the immune system's response. Sarepta said it was cooperating with the Food and Drug Administration, which would have to sign off on any changes to the product's use.
Elevidys received expedited approval despite concerns from some FDA scientists about its effectiveness in treating Duchenne's. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older regardless of whether they are still able to walk. Previously, it was only available for younger patients who were still walking.
Shares of the Cambridge, Massachusetts–based company fell more than 42 percent to close at about 21 in trading. Wall Street analysts speculated that FDA officials, including new vaccine chief Dr. Vinay Prasad, might impose more restrictions on the drug or even block its use. Prasad has been highly critical of the therapy since its approval under the agency's previous leadership.
'Now with two deaths reported in this segment of the market, it seems incrementally more possible that the FDA could step in and remove the therapy from the market in non-ambulatory patients,' said Leerink Partners analyst Joseph Schwartz in a Sunday research note.
Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin into patient cells. It costs $3.2 million for a one-time infusion.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Al Arabiya
2 hours ago
- Al Arabiya
Sprains, strains and acl tears: What to know about some of nfl players' most common injuries
Some of the most important players on NFL teams are those that might not necessarily start the season on the field. Depth is crucial during a rigorous 17-game regular-season schedule that's preceded by a month of training camp practices in hot conditions as players try to make teams' 53-man active rosters. Injuries can play as big a role in an NFL teams' successes or failures as the best game plans. So being able to navigate injuries and ailments to key players could make all the difference between a team seeing its season sink or making the playoffs. Here's a look at some of the most common injuries suffered by NFL players throughout the season: Achilles tendon tears: The Achilles is the longest and strongest tendon in the body and stretches from the heel to the calf. It's a springy band located behind the ankle and just above the heel that helps players push off their feet, jump, and accelerate. Due to overuse or excessive force placed on the tendon, it can tear or rupture. Surgery is typically necessary to reconnect the ends, sidelining a player for several months because of the extensive rehabilitation needed. ACL/PCL/MCL/LCL tears: The anterior cruciate, posterior cruciate, medial collateral, and lateral collateral ligaments are all located in the knee and serve different purposes. The ACL connects the thigh bone to the shin bone in the front of the knee. While a sprain could sideline a player for a few weeks, a tear can end a season. The PCL is located behind the ACL, crisscrossing it to form an X in the center of the knee. The MCL connects the thigh bone to the shin bone on the inner side of the knee while the LCL connects the thigh bone to the top of the lower leg or fibula and is located on the outer side of the knee. Concussions: A concussion is a brain injury caused by a hit to the head or a sudden movement that causes the head and brain to shake violently. Symptoms may include headaches, neck pain, nausea, dizziness, and feeling sluggish. The NFL has been regularly testing and upgrading helmets for players to help reduce the risk of concussions. Eight position-specific helmets for quarterbacks and linemen were approved by the NFL and the NFL Players Association last year. Several players also have worn Guardian Caps, soft protective helmet covers that the NFL authorized players to use during games last season in an effort to reduce head injuries. Chronic traumatic encephalopathy, or CTE, is a degenerative brain disease that has been linked to concussions and can only be diagnosed posthumously. It can cause memory loss, depression, and violent mood swings. Hamstring injuries: The hamstring is a group of four muscles that run along the back of the thigh stretching from the hip to the knee and they help a person bend their leg at the knee. These injuries vary in severity and in turn their timetable in sidelining a player. A mild pull of one of the muscles commonly referred to as a Grade I injury can sideline a player for a few days to a couple of weeks and can be a lingering condition if not sufficiently rested and healed. A Grade II hamstring injury involves a partial tear, while a Grade III injury is a complete tear of the hamstring that could require surgery and is likely season-ending. High ankle sprains: When a player suffers a high ankle sprain, the ligaments above the ankle – which connect the tibia to the fibula – are affected rather than the ligaments outside the ankle in a low ankle sprain. High ankle sprains take much longer to recover from – six to eight weeks and sometimes longer – than a classic ankle sprain, which might sideline a player for several days to a couple of weeks. Hip pointers: The injury could sideline a player for a week or a couple of months depending on the severity. A hip pointer is bruising in the pelvis and abdomen area usually caused by blunt force such as a hard tackle. The bleeding can affect several other muscles in the area making it difficult to run or even walk. Lisfranc injuries: A serious foot injury that can be career-threatening because of its complexity. A Lisfranc sprain or fracture is an injury in the middle of the foot in which at least one (or sometimes all) of the small bones (or metatarsals) is broken or the ligaments that support the foot in that area are torn. Even a minor sprain not requiring surgical repair could take six to eight weeks to heal. Meniscus tears: The meniscus is a crescent-shaped rubbery disk of cartilage that serves as a shock absorber on the inside and outer edges of the knee. Both help a person balance weight across the knee. When a meniscus is torn, the knee can lock up and swell. A minor tear can be treated with rest but a severe tear could require surgery that may sideline a player for several weeks or months. Oblique strains: An oblique strain involves the muscles on the side of the body between the ribs and pelvis. This type of injury can occur when a player takes a hard hit to the waist area or from overuse or sudden use of the muscles – for instance, a quarterback throwing a pass or a defensive back turning to defend a receiver. Oblique strains are usually treated with rest and could take a few weeks to heal or could become a lingering issue otherwise. Patellar tendon injuries: The patellar tendon allows a person to straighten a leg by acting with the quadriceps. Technically it's a ligament because it connects the kneecap to the shin bone. Complete tears or ruptures often need to be surgically sewn back together and recovery is typically at least four to six months. Plantar fasciitis: An injury that affects the bottom of the foot and can lead to intense heel pain. Plantar fasciitis occurs when the ligament supporting the arch of the foot – the plantar fascia – is strained and worsens when small tears develop in the ligament. The injury can sap players of speed while they deal with it. Rest, icing of the arch, and finding new footwear are among typical treatments. It can linger for months. Turf toe: An extremely painful injury that can sideline players for months. It occurs when the ligaments under the joint of the big toe are sprained or ruptured as a result of the toe being hyperextended. The injury makes it extremely difficult to push off and cut while running.
Al Arabiya
3 hours ago
- Al Arabiya
8 children taken to hospitals after seizure-like symptoms at Harvard Square church concert
Eight children at a church near Harvard University, where a French youth choir was holding a concert, suffered seizure-like symptoms and were taken to hospitals, officials said. The symptoms were not life-threatening, the Cambridge Fire Department said in a news release. About 70 other people in attendance at the concert Tuesday evening at St. Paul's Parish in Harvard Square were not affected. Crews first received a call about a child suffering from a seizure. When firefighters arrived, the child was sitting outside of the church but was not actively having a seizure, Fire Chief Thomas Cahill said. That quickly escalated into seven other people having seizure-like symptoms, Cahill told WCVB-TV. The department's hazmat team completed a thorough survey of the St. Paul buildings, utilizing several air sampling meters to ensure that no hazardous conditions were present, the news release said. Results were negative, and the buildings were ventilated. St. Paul's Facebook Page said a French youth choir, the Chœur d'Enfants d'Île-de-France, was offering a free concert at the church on Tuesday night as part of its 2025 US tour. Founded in 1970, the youth choir has traveled extensively and performed with some of the world's top conductors and soloists, St. Paul's said. The concert will feature an exciting mix of sacred and secular repertoire, as well as popular French songs. Messages seeking comment were emailed to St. Paul's and to the Harvard Catholic Center, the Catholic chaplaincy for Harvard University students and other academic institutions in the area.
Al Arabiya
a day ago
- Al Arabiya
Tennessee judge gives state leeway on deactivating inmate's heart-regulating device before execution
Nashville, Tenn. – A Tennessee judge ruled on Tuesday that the state can deactivate a death-row inmate's implanted heart-regulating device at a hospital on the morning of his execution, rather than bringing a doctor or technician into the execution chamber. The ruling comes after Deputy Attorney General Cody Brandon told the judge they could not comply with an earlier order requiring Byron Black's implanted cardioverter-defibrillator to be turned off just prior to the injection of a lethal dose of pentobarbital on Aug. 5. In the Friday order, Davidson County Chancery Court Judge Russell Perkins agreed with Black's attorneys that not deactivating the device could cause Black to suffer needlessly as it continuously shocks him in an attempt to restore his heart's normal rhythm during the execution. At a Tuesday hearing on the issue, Brandon said Black's physicians at Nashville General Hospital are unwilling to come to the execution chamber. That means the Friday chancellor's order is in effect an order to stay the execution, which only the Tennessee Supreme Court has the power to do, he argued. Brandon asked the judge to either overturn his Friday order or allow the Tennessee Department of Correction to take Black to the hospital for the deactivation on Aug. 4. Kelley Henry, who represents Black, argued that the state should not be allowed to deactivate the device prematurely because doing so could result in Black's death while he still has the possibility of a last-minute reprieve. 'On August 4 we will still be in other courts seeking a stay of execution,' Henry said. She also said state officials had not really tried to find a doctor willing to come to the prison. She first brought the issue up with the Tennessee Department of Correction in early June, but it was not until the Friday order that they began to look into it, and then they only contacted Nashville General Hospital. 'It's like when you tell a teenager to clean up their room and they pick up one sock,' she said. The implantable cardioverter-defibrillator is a small battery-powered electronic instrument surgically implanted in the chest that serves as a pacemaker and an emergency defibrillator. Perkins noted in court on Tuesday that during a two-day hearing last week on the issue, a doctor testifying for Black said that deactivating the device is quick and doesn't require surgery, only a handheld machine. Meanwhile, the state did not present any testimony at the time that deactivation would be an administrative or logistical burden. In modifying his own order on Tuesday, Perkins wrote that 'it is obvious the state has taken only minimal steps to comply with his Friday order.' However, Perkins said he also wants to ensure that his ruling can't be construed as an attempt to interfere with the execution. 'I've lost sleep over this because I want to do the right thing,' Perkins said at the hearing. 'I want to honor the memories of these victims while also making sure the execution is properly carried out.' Black was convicted in the 1988 shooting deaths of girlfriend Angela Clay, 29, and her two daughters, Latoya, 9, and Lakeisha, 6. Prosecutors said Black was in a jealous rage when he shot the three at their home. At the time, Black was on work-release while serving time for shooting and wounding Clay's estranged husband. 'Had she not been killed, Lakeisha would be in her 40s today,' Brandon told the judge on Tuesday. 'Justice has waited 37 years. It should not wait any longer.' Black's motion related to his heart device came within a general challenge he and other death row inmates filed against the state's new execution protocol. The trial isn't until 2026.
