Treatments & Care

The information provided on this website is designed to support, not replace, the relationship that exists between a patient/site visitor and their physician.

Treatment for Degos Disease

Currently, no established standard of care exists for Degos disease due to its ultra-rare nature. Treatment options are still emerging, and we aim to provide general guidance and resources to help you stay informed.

Anifrolumab, Eculizumab, and Treprostinil have all been utilized in many cases as off-label treatments, offering hope for symptom management. You and your doctor can find more detailed information in this article from The Lancet on Anifrolumab. Additional case reports of these agents are in process of publication.

For further updates on treatments and drug access, we will update this page as more studies are done. Please contact one of the following physicians for further guidance on what treatments would be best for your patient.

For specific questions or case consultations, please reach out to one of these specialists:

The use of Anifrolumab, Eculizumab, and Treprostinil for Degos disease are considered "off-label," which often makes timely access to these medications a challenge. Below are some key strategies to help improve or expedite drug access:

Drug Access

  • Insurance/Coverage: Insurance companies may deny initial requests for off-label treatments. Writing a letter to the insurance company outlining the necessity of the treatment can sometimes lead to approval. Be prepared for a lengthy appeal process, as multiple attempts may be required

  • Compassionate Use: In cases where standard avenues fail, working with your physician to request Compassionate Use access may be an option

  • Advocacy: It's important to advocate for yourself by staying informed and persistent in requesting access to treatment

For a more detailed look at global challenges related to rare disease treatment, see the article Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges, which outlines the barriers to drug access in different regions. Table 1 in this article highlights challenges related to diagnosis, treatment access, quality of life, and financial protection across the globe.

Access to rare disease treatments varies significantly based on geographic location. Factors such as healthcare infrastructure, insurance coverage, and economic resources can impact a patient's ability to receive timely treatment. The article mentioned above provides further insights into these issues.

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Patient Stories

Hear firsthand accounts of resilience and hope from individuals like you.

What is Degos Disease?

Learn more about Degos disease and what it means to have a rare disease.

References

  1. Adachi, T., El-Hattab, A. W., Jain, R., Nogales Crespo, K. A., Quirland Lazo, C. I., Scarpa, M., Summar, M., & Wattanasirichaigoon, D. (2023). Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges. International Journal of Environmental Research and Public Health, 20(6), 4732. https://doi.org/10.3390/ijerph20064732

  2. Clinical manifestations and treatment outcomes in Degos disease: A systematic review. https://onlinelibrary.wiley.com/doi/10.1111/jdv.17311

  3. Gene identified and new therapy developed for a young patient with a rare disease: Dr. Lena-Luise Becker receives Dr. Holger Müller Prize 2022. https://www.care-for-rare.org/verleihung-des-11-dr-holger-mueller-preises-dr-lena-luise-becker-fuer-forschungsarbeit-ausgezeichnet/