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☆ Opinion | Three Mothers’ Plea to the F.D.A.: Save Our Children
▼ + stars: | 2024-02-28 | by ( ) www.nytimes.com time to read: +1 min

To the Editor:Re “Why Can’t More Children Get the Treatment That Saved My Son’s Life?,” by Elizabeth Currid-Halkett (Opinion guest essay, Feb. 24):Our three children, ages 5 and 7, battle a rare, relentless and ultimately fatal disease called cystinosis. We recently found hope in the initial phase of a gene therapy clinical trial that was shown to be safe and yielded very promising results — a therapy that could one day save our children’s lives. Our biggest fear is that it will not be accessible to them or others in desperate need. We applaud Dr. Marks’s wisdom and perspective. reviewers’ initial rejection is a cautionary tale of how patient access to lifesaving therapies could be impeded by a narrow interpretation of efficacy.

Persons: Elizabeth Currid, Currid, Peter Marks, “ Organizations: Biologics

☆ Gene Therapies Could Transform Rare Diseases. Are We Holding Them Back? - The New York Times
▼ + stars: | 2024-02-19 | by ( Elizabeth Currid-Halkett | ) www.nytimes.com time to read: +1 min

On Jan. 8, 2020, as I was parking my car, I got a long-awaited phone call from one of my son’s doctors. She informed me that our 7-month-old son, Eliot, had Duchenne muscular dystrophy, a fatal neuromuscular disease. I can see my neighbor walking up her steps with groceries, a leaf falling, oblivious to the devastation below. It is caused by a genetic mutation on the X chromosome, thus the disease almost exclusively affects boys (one in 3,300). lose muscle mass and thus the ability to do basic things like run and walk.

Persons: Eliot, Joan Didion, D.M.D, Jerry Mendell Organizations: Nationwide Children’s Hospital, D.M.D Locations: Columbus , Ohio

In the hills north of Kansas City, Mo., a small abbey has become a destination for Catholics hoping to see a miracle. Sept. 9, 2023In life, Sister Wilhelmina Lancaster was known to her fellow nuns for her devotional poetry, her sense of humor and her fierce piety. “I’m Sister Wil-hel-mina,” she was known to say. When they opened the coffin, expecting to find bones that could be easily cleaned and placed in a new box, they instead found what looked and even felt remarkably like Sister Wilhelmina herself. Her face was recognizable, even after years in a damp coffin, and the sisters said that her beloved habit was “immaculate.”

Persons: Sister Wilhelmina Lancaster, “, Wil, ”, Sister Wilhelmina, Ephesus, Mary , Queen, Wilhelmina Organizations: Benedictines, Kansas City . Locations: Kansas City, Mo, hel, mina, Kansas

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