Parents of Children with Orphan Diseases Need Help Too in America

What a sad-sounding name for this group of diseases, and an even sadder reason that this designation was assigned to them. Orphan diseases are rare, mostly genetic, diseases that don’t have cures,and many times not even treatments. Although there are more than 7000 of them, each individual disease has so few cases that it doesn’t justify the time or money necessary to invest in research and development for drugs for each illness. In other words, no one wants to “adopt” them to develop treatments, so they are “orphans”. This moniker was given by Congress with the passing of the Orphan Drug Act of 1983, which was designed to offer incentives to drug companies to develop treatments for rare diseases. Efforts to improve the research and development of new treatments is handled by the Food and Drug Administration (FDA) and some progress is being made.

Different countries have their own statistical criteria for which ones will be named an orphan disease, but it is estimated that one in every fifteen people, or approximately 400 million worldwide, have been diagnosed with one, and more are continually being discovered. About 30 % of these children die before the age of five. Some of the diseases you may have heard of, such as amyotrophic lateral sclerosis(ALS, or Lou Gehrig’s Disease), chronic lymphocytic leukemia (CLL), and hemophilia, and others may be completely foreign to you. Although each one is different, there is one thing that these children have in common: their parents need help, too.

Studies show consistent responses when parents were asked about the difficulties they face.Orphan diseases are hard to diagnose, and it sometimes takes years to get the correct one,creating anxiety and frustration. One very concerning issue is that many healthcare professionals have never seen, or sometimes even heard of, some of these unusual entities, makingthem ill-equipped to diagnose them or to determinethe child’s plan of care. Once the diagnosis has finally been made, parents’ heads are swirling with confusion, uncertainty, concern, and fear, and they must live with the knowledge that they will, in all probability, lose their child. A genetics professional can help them with determining hereditary factors and with future decisions.

Parents may face a lack of support on several fronts. Financially, many of these families are struggling. With little-known diseases come little-known or experimental treatments, many of which may not be covered by standard medical plans. Not only must they pay for costly treatments out of pocket, but they may be forced to reduce working hours or hire help, both of which further diminish their resources. Anxiety and depression are common, and psychological help is expensive.Parents are already spending money trying to provide quality care for their child, so there may not be enough left to provide the family with ongoing psychological support. Additionally, there may be a dearth of qualified caregivers, depending on the child’s needs

These parents are unable to socialize as before or even to live normal lives, so they can face isolation. It’s not uncommon for them to lose friends or even to lose contact with family members. Friends and family may be empathetic, but sometimes, they just don’t know what to say or do, so they distance themselves.

These families need our help. Do you know anyone living with this type of special needs child? Give them an ear; they need to vent. Cook them a meal; they’re probably exhausted. Arrange child care for a night or weekend; they need to get away occasionally. Offer to shop, or just ask them what you can do, and then do what you can.


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