Update: Twins who were face of controversial rare disease treatment have died

Science | AAAS | 7/18/2019 | Staff
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*Update, 18 July, 4:30 p.m.: The Hempel twins, Addi and Cassi, died on 4 July. They were 14 years old. “They passed away within minutes of each other and we are filled with so much sorrow,” their mother, Chris Hempel, wrote in an email to ScienceInsider.

She wrote that her daughters had been admitted to Renown Regional Medical Center in Reno, Nevada, on 29 and 30 June, after they developed labored breathing and high temperatures. The cause, it emerged, was an aggressive virus that had invaded their lungs. Although Niemann-Pick type C (NPC) was not on their death reports, “Certainly their underlying NPC disease was a contributing factor,” Hempel wrote. “[T]heir pulmonary systems were already weakened by the NPC.”

Hempel - Husband - Hugh - Use - Sugar

Hempel and her husband, Hugh, pioneered the experimental use of a sugar molecule, 2-hydroxypropyl-β-cyclodextrin, as a treatment for the rare genetic disease, winning a compassionate use approval from the U.S. Food and Drug Administration for its use in the twins. In January 2018, they sued several companies involved in developing a commercial treatment; their allegations include breach of contract, theft of trade secrets, and unjust enrichment. The Hempels’ lawyer on 11 July filed a request for a delay in the proceedings in U.S. District Court in Maryland in order to allow the Hempels time to grieve. But Chris Hempel wrote that they plan to press on “vigorously” with that lawsuit.

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Here is our previous story from 9 November 2018:

Treatment - Niemann-Pick - Type - C - NPC

A treatment for Niemann-Pick type C (NPC), an extremely rare and ultimately fatal neurodegenerative disease, performed no differently than placebo in a pivotal trial in 56 children and youths, its corporate sponsor announced on Tuesday. Perplexingly, though, the disease did not progress in either the treatment or placebo groups during the 1-year...
(Excerpt) Read more at: Science | AAAS
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