Clinical Brief: Updates in Managing Rare Diseases: Tips for the Practicing Dermatologist

In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people in the US. While each rare disease may not seem to affect so many people, there may be as many as 7,000 rare diseases affecting an estimated 25-30 million Americans. More specifically, there over 1,000 rare skin diseases that are often an extreme burden to the collective many of patients affected by these conditions.

Epidermolysis bullosa (EB), a group of rare genetic skin disorders that result in fragile skin and painful blister formation, currently have no cure. While this disease has been found to affect only 2.12 and 1.49 cases per 1 million live births, the burden of this disease is staggering. Prurigo nodularis (PN), a chronic inflammatory skin disease, is thought to affect approximately 72 per 100,000 adults in the US. While corticosteroids have been employed to help manage the symptoms, there is no FDA approved treatment for this burdensome disease. Pemphigus vulgaris (PV), a rare group of autoimmune diseases that causes blistering on the skin and mucous membranes throughout the body, has an incidence rate estimated between 0.5 and 3.2 cases per 100,000 population. PV is the only one of these rare diseases that has an FDA approved treatment, but clinicians may not yet know strategies for treatment or patient selection.

Therefore, it is critically important that clinicians are aware of emerging therapies in clinical development for these rare skin diseases and how to use them for optimal individualized treatment. This clinical brief will provide a succinct overview of these rare skin diseases and help prepare clinicians to potentially utilize these therapies in their own practice.

Target Audience

This activity is designed to target US-based physicians, nurses and professional staff members of medical practices specializing in dermatology as well as nurses, nurse practitioners, physician assistants, and pharmacists.

Learning Objectives

  • Identify diagnostic criteria for rare dermatologic diseases such as epidermolysis bullosa (EB), prurigo nodularis (PN), and pemphigus vulgaris (PV)
  • Review current and new treatments for EB, PN, and PV, including relevant safety and efficacy date
Course summary
Available credit: 
  • 0.25 AAPA Category I CME
  • 0.25 AMA PRA Category 1 Credit
  • 0.25 ANCC
  • 0.25 Participation
Course opens: 
Course expires: 

M. Peter Marinkovich, MD | Scholars | Harrington Discovery Institute at  University Hospitals

M. Peter Marinkovich, MD

Associate Professor of Dermatology

Stanford Hospital

Redwood City, CA




M. Peter Marinkovich, MDNothing to disclose 


Jointly provided by the AKH Inc., Advancing Knowledge in Healthcare and LiVDerm.

Introducing Featured Member AKH Inc. Advancing Knowledge in Healthcare

Estimated time to complete activity: 15 minutes

Commercial Support

This activity is supported by an educational grant from Amryt Pharma.

Physician Continuing Medical Education

Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by AKH Inc., Advancing Knowledge in Healthcare and Tarsus Cardio dba LiVDerm. AKH Inc., Advancing Knowledge in Healthcare is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.


AKH Inc., Advancing Knowledge in Healthcare designates this enduring material activity for a maximum of 0.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.


0.25 ANCC contact hours

Physician Assistant

AAPA Category 1 CME logo | Clinical Preceptors | Physicians Assistant  Program | Presbyterian College | Clinton SC - Academics

AKH Inc., Advancing Knowledge in Healthcare has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for .25 AAPA Category 1 CME credits. Approval is valid until October 20, 2023. PAs should only claim credit commensurate with the extent of their participation.


It is the policy of AKH Inc. to ensure independence, balance, objectivity, scientific rigor, and integrity in all of its continuing education activities. The author must disclose to the participants any significant relationships with ineligible companies whose products or devices may be mentioned in the activity or with the commercial supporter of this continuing education activity. Identified conflicts of interest are mitigated by AKH prior to accreditation of the activity. AKH planners and reviewers have no relevant financial relationships to disclose.





M. Peter Marinkovich, MDN/ANothing to disclose

Dorothy Caputo, MA, BSN, RN - Senior Director of Continuing Education & Compliance


Nothing to disclose

Michelle Bielarski, RNN/ANothing to disclose

LiVDerm Planners and Reviewers


Nothing to disclose

AKH Planners and Reviewers


Nothing to disclose

All of the relevant financial relationships listed for these individuals have been mitigated.


This course is designed solely to provide the healthcare professional with information to assist in his/her practice and professional development and is not to be considered a diagnostic tool to replace professional advice or treatment. The course serves as a general guide to the healthcare professional, and therefore, cannot be considered as giving legal, nursing, medical, or other professional advice in specific cases. AKH Inc. specifically disclaim responsibility for any adverse consequences resulting directly or indirectly from information in the course, for undetected error, or through participant's misunderstanding of the content.

Disclosure of Unlabeled Use and Investigational Product

This educational activity may include discussion of uses of agents that are investigational and/or unapproved by the FDA. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Available Credit

  • 0.25 AAPA Category I CME
  • 0.25 AMA PRA Category 1 Credit
  • 0.25 ANCC
  • 0.25 Participation
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Other additional software may be required such as PowerPoint or Adobe Acrobat Reader.