After 44 days, Kendric Cromer, 12, left the hospital. While his family feels fortunate that he was the first to receive a treatment, their difficult experiences hint at what others will be up against.Kendric Cromer, 12, left Children’s National Hospital in a wheelchair on Wednesday, wearing a T-shirt and cap printed with designs from the anime series “Naruto” and a black face mask. Staff lined the hallway, cheering and waving noisemakers. He had just become the first patient to receive a gene therapy for sickle cell since it was approved — a therapy that is expected to free him from the ravages of the disease.After 44 days in the hospital, he was a bit dazed.“I thought I would have sickle cell for the rest of my life,” he said. The disease had deprived him of his childhood, making everyday activities, like playing basketball or riding a bike, impossible because they could bring on searing pain, often resulting in hospitalizations.But despite the celebratory atmosphere, Kendric and his parents are still shuddering over what they endured during his hospital stay.Nothing, absolutely nothing — not all the discussions with doctors, not all of their reading and highlighting of texts, not the 13-page consent form that included organ damage and even death as possible outcomes — prepared them for what he would go through.About 100,000 people in the United States have sickle cell disease. For the 20,000 or so with the most severe disease, gene therapy may be their only hope of living a normal life. The disease is caused by a mutation in hemoglobin genes that leads to crescent-shaped red blood cells, which tend to get stuck in blood vessels, causing episodes of excruciating pain. The blockages can damage organs, cause strokes and shorten lives.Until recently, most saw no way out.Then, last December, the Food and Drug Administration approved a $3.1 million sickle cell gene therapy by Bluebird Bio of Somerville, Mass., and a $2.2 million treatment by Vertex Pharmaceuticals of Boston. That potentially gives patients like Kendric, if their insurance will pay for the therapy, a path to a life that is not shadowed by the ravages of the disease.We are having trouble retrieving the article content.Please enable JavaScript in your browser settings.Thank you for your patience while we verify access. If you are in Reader mode please exit and log into your Times account, or subscribe for all of The Times.Thank you for your patience while we verify access.Already a subscriber? Log in.Want all of The Times? Subscribe.

There was supposed to be a special party for Kendric Cromer, 12, last Wednesday, but it had to be postponed because he was too groggy to celebrate.It was meant to mark the first day of his new life — the day he became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood.On Sept. 11, despite the excitement of the moment, Kendric was unable to keep his eyes open as he lay in his hospital bed at Children’s National Hospital in Washington because of the drugs he had been given in preparation for his treatment.His life with the disease has been punctuated by episodes of excruciating pain, requiring days in the hospital as doctors tried to control it. Sickle cell eroded his hip bones. It prevented him from riding a bike or playing soccer or even going outside when the temperature was below 55 degrees Fahrenheit because cold often brought on intense pain.Now he could see a future — in a month or so — without pain from sickle cell.“I can’t wait to start my new life,” he told his mother, Deborah Cromer.His disease is caused by an inherited genetic mutation that leads to blood cells that form crescent shapes — sickles — instead of discs. Trapped in blood vessels and organs, the cells cause damage and pain. Gene therapy fixes that problem by giving the patient a new, normal hemoglobin gene.We are having trouble retrieving the article content.Please enable JavaScript in your browser settings.Thank you for your patience while we verify access. If you are in Reader mode please exit and log into your Times account, or subscribe for all of The Times.Thank you for your patience while we verify access.Already a subscriber? Log in.Want all of The Times? Subscribe.

A 12-year-old boy in the Washington, D.C., area faces months of procedures to remedy his disease. “I want to be cured,” he said.On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition.For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments.For a lucky few, like Kendric, the treatment could make possible lives they have longed for. A solemn and shy adolescent, he had learned that ordinary activities — riding a bike, going outside on a cold day, playing soccer — could bring on episodes of searing pain.“Sickle cell always steals my dreams and interrupts all the things I want to do,” he said. Now he feels as if he has a chance for a normal life.Near the end of last year, the Food and Drug Administration gave two companies authorization to sell gene therapy to people with sickle cell disease — a genetic disorder of red blood cells that causes debilitating pain and other medical problems. An estimated 100,000 people in the United States have sickle cell, most of them Black. People are born with the disease when they inherit the mutated gene for the condition from each parent.The treatment helped patients in clinical trials, but Kendric is the first commercial patient for Bluebird Bio, a Somerville, Mass., company. Another other company, Vertex Therapeutics of Boston, declined to say if it had started treatment for any patients with its approved CRISPR gene-editing-based remedy.We are having trouble retrieving the article content.Please enable JavaScript in your browser settings.Thank you for your patience while we verify access. If you are in Reader mode please exit and log into your Times account, or subscribe for all of The Times.Thank you for your patience while we verify access.Already a subscriber? Log in.Want all of The Times? Subscribe.