Families Find New Strategies To Fight Children’s Rare Diseases
Many of these diseases don’t get large amounts of research funding, so parents are forced to try to find alternatives. The New York Times and The Washington Post look at some of these efforts.
The New York Times:
Flicker Of Hope For Children With Rare And Devastating Disease
Once a year, Crystal and Jonathan Bedford drive 1,000 miles from their home in Texas to rural Alabama, their three children in tow. Beside a wooded lake, they huddle with other families whose children have the same extremely rare genetic disorder that their 5-year-old daughter, Marley, has. The disease, rhizomelic chondrodysplasia punctata, is a painful form of dwarfism, usually accompanied by severe intellectual disability and respiratory problems. There is no cure, and children with RCDP, as it is known, rarely survive into adolescence. ... But this year was different. A biotech executive from Canada had come to discuss a potential treatment being developed by his company and the possibility that the children could be part of a clinical trial next year. It seemed too good to be true. (Goodnough, 9/6)
The Washington Post:
Family Carves Out Its Own Path In Fight Against 4-Year-Old’s Rare Disease
Arturito [Estopiñan] was 1 when he was diagnosed with a disease caused by a disruption in the mitochondria, a part of the cell that supports growth. Only 83 such cases have ever been recorded. Doctors told his parents, Arturo and Olga, that there was no treatment and that he would die soon. ... Today, Arturito is enduring with the help of an experimental drug that only a handful of people in the world are taking. ... The Estopiñans’ experience illustrates how families struggle to find help fighting diseases that don’t attract a huge amount of research or specialized funding. It also shows how determined parents can forge a path for other families encountering similar obstacles. (Gebelhoff, 9/7)
Another article examines how many patients are turning to crowdfunding.
The Sacramento Bee:
Rise In Crowdfunding Lets Patients Seek Help For Medical Treatment
Internationally, medical crowdfunding campaigns on GoFundMe have jumped from just over 8,000 in 2011 to more than 740,000 this year. Some people donate a few dollars, some a few hundred. And patients’ loved ones often use social media to spread the word. (Kuang, 9/7)