Future Treatment Directions
Better treatments for AHC may become a reality as we gain a better understanding of the disease, but progress to date has been extremely slow. Clinical trials to better assess the effects of medications on symptoms in AHC are desperately needed.
The relative rarity of this disorder makes it critical that families and physicians worldwide work together to gain a better understanding of the disease, and to collaborate in pursuing genetic studies and clinical trials. In the meantime, a proactive management strategy and a network of supportive health care professionals that you can rely on in a crisis situation is critical.
Good care is unlikely to be gained from the emergency room physician who has never heard of AHC. Rather, it is best to develop a proactive care plan, including contingent plans for emergencies. Participation in clinical studies and networking with other parents and support groups can help accelerate progress. Participation in studies is vital to the community to help demonstrate the effectiveness of potential treatments and to better understand this disease.
For more information about family support organizations for AHC, visit www.ahckids.org
To find out about participating in clinical studies on AHC, or to join our AHC registry, contact us 801-587-9260, or email Matthew Sweney, M.D., AHC research fellow, Sweney@genetics.utah.edu.