Epidermolysis bullosa is a rare genetic condition that causes the skin to become extremely fragile and prone to blistering from even minor friction or trauma. Treatment focuses on protecting the skin, managing painful blisters, preventing infections, and addressing complications affecting multiple body systems.

How Treatment Helps People Living with Fragile Skin

When someone is diagnosed with epidermolysis bullosa, also known as EB, the main goals of treatment center on improving quality of life and preventing serious complications. Because there is currently no cure for this condition, medical care focuses on protecting the delicate skin, reducing pain, promoting wound healing, and supporting nutrition when blisters affect the mouth or digestive system. The severity of EB varies widely from person to person, which means treatment plans must be tailored to each individual’s needs and the specific type of EB they have.^[1][2]

The approach to managing EB depends heavily on which form of the disease a person has and how severely it affects them. Some people experience blistering mainly on their hands and feet, while others develop widespread blisters across their entire body and even inside their mouth, throat, or digestive tract. Treatment decisions also take into account the person’s age, overall health, and how well their body responds to different therapies. Medical teams work closely with families to develop comprehensive care plans that address both immediate needs and long-term health.^[6]

Standard medical treatments approved by health authorities provide the foundation of care for people with EB. At the same time, researchers around the world are investigating new therapies in clinical trials, which are carefully controlled research studies that test whether new treatments are safe and effective. These experimental approaches include gene therapies, protein-based treatments, and innovative medications that target the underlying causes of skin fragility. Participation in clinical trials offers some patients access to cutting-edge treatments that may not yet be widely available.^[13][15]

Standard Treatment Approaches for Daily Care

The cornerstone of EB treatment is meticulous wound care, which often requires several hours each day. Families learn specific techniques for managing blisters as soon as they appear. One essential practice involves carefully puncturing new blisters with a sterile needle to drain the fluid inside. This prevents blisters from expanding and creating larger, more painful wounds. The skin covering the blister is typically left in place to act as a natural protective barrier for the raw tissue beneath.^[11][17]

Protecting wounds and preventing infection requires specialized bandages and dressings. Healthcare providers recommend using non-stick dressings that will not adhere to fragile skin when removed. These dressings must be changed regularly, often daily, to keep wounds clean and promote healing. Petroleum-based ointments and antibiotic creams help keep the skin moist and reduce the risk of infection. Families must avoid using regular adhesive bandages or tape, as these can tear the skin when removed.^[9][10]

A significant advancement came in 2023 when health authorities approved birch triterpenes topical gel, sold under the brand name Filsuvez, for treating wounds in people aged 6 months and older who have certain forms of EB. This gel, made from an extract of birch bark, has been shown in clinical studies to help wounds heal more quickly. It represents the first topical medication specifically approved for EB wound care. Patients apply the gel directly to wounds as directed by their healthcare provider.^[13][16]

Pain management is another critical component of standard care. People with EB often experience significant pain from their blisters and open wounds. Doctors may prescribe medications to control pain, ranging from over-the-counter options for mild cases to stronger prescription pain relievers for more severe forms of the disease. Managing itching is also important, as scratching can create new blisters. Healthcare providers may recommend medications that reduce itching and help patients feel more comfortable.^[9][16]

Preventing skin damage requires constant attention to everyday activities. Families learn to modify how they handle, dress, and care for someone with EB. This includes choosing soft, loose-fitting clothing made from natural fabrics like cotton, selecting shoes that fit properly without rubbing, and avoiding activities that create excessive friction on the skin. Keeping the body cool is important because sweating and heat can trigger more blistering. Healthcare teams provide detailed guidance on protecting the skin while still allowing people with EB to participate in daily life as much as possible.^[11][17]

Nutrition support becomes necessary when blistering affects the mouth, throat, or esophagus—the tube that carries food from the mouth to the stomach. Painful blisters in these areas make eating difficult and can lead to poor nutrition and inadequate growth, especially in children. Healthcare providers may recommend soft foods, nutritional supplements, and special feeding techniques. In severe cases where eating becomes too painful or impossible, doctors may surgically place a feeding tube directly into the stomach to ensure adequate nutrition. Some patients require surgery to widen the esophagus if repeated blistering and scarring cause it to narrow.^[9][11]

When infections develop in open wounds, they must be treated promptly with antibiotics—medications that kill bacteria. Signs of infection include increased redness and warmth around wounds, pus or cloudy drainage, fever, or wounds that fail to heal. Doctors may prescribe antibiotic pills or creams depending on the severity and location of the infection. In severe cases, patients may need to be hospitalized for intravenous antibiotics delivered directly into the bloodstream.^[11][16]

People with EB require care from multiple medical specialists working together as a team. This interdisciplinary approach typically includes dermatologists who specialize in skin conditions, nutritionists who address dietary needs, physical therapists who help maintain movement and prevent joint stiffness, dentists who manage dental problems common in EB, and nurses who coordinate care and provide hands-on treatment guidance. Social workers help families navigate insurance issues and connect with support resources. Regular follow-up appointments allow the team to monitor for complications and adjust treatment as needed.^[6][11]

Surgical interventions may become necessary to address specific complications. Beyond feeding tubes and esophageal widening, some patients require surgery to separate fingers or toes that have fused together due to repeated blistering and scarring. In severe cases where EB causes significant scarring and deformities that limit movement, reconstructive surgery may help restore some function. Additionally, because certain forms of EB significantly increase the risk of developing squamous cell carcinoma, a type of skin cancer, any suspicious skin areas must be removed surgically and examined.^[9][16]

Innovative Treatments Being Tested in Clinical Trials

The landscape of EB treatment has been transformed in recent years by the development of gene therapy, which aims to correct the genetic defects that cause the disease. In 2023, the United States Food and Drug Administration approved the first topical gene therapy for EB called beremagene geperpavec, marketed as Vyjuvek. This groundbreaking treatment is specifically designed for people with dystrophic epidermolysis bullosa, or DEB, a form of the disease caused by mutations in the gene responsible for producing type VII collagen, a protein essential for holding skin layers together.^[13][16]

Beremagene geperpavec works by delivering a functional copy of the COL7A1 gene directly to skin cells through a modified virus that cannot cause disease. When applied topically to wounds, the therapy enters both the outer skin cells called keratinocytes and the deeper cells called fibroblasts. Once inside these cells, the corrected gene instructs them to produce normal type VII collagen protein. This collagen then forms anchoring fibrils, the tiny structures that attach the outer and inner layers of skin together, preventing blisters from forming. The approval of this therapy came after a Phase 3 clinical trial demonstrated its safety and effectiveness in helping wounds heal.^[16]

Another revolutionary gene therapy approved in April 2025 is prademagene zamikeracel, known commercially as Zevaskyn. This treatment takes a different approach by using the patient’s own skin cells. Doctors remove a small sample of the patient’s skin through a biopsy, then modify these cells in a laboratory to correct the genetic defect. The corrected cells are grown into thin sheets of skin that can be transplanted back onto the patient’s wounds. Because the therapy uses the patient’s own cells—making it an autologous treatment—there is minimal risk of rejection. This cell-based gene therapy is approved for both children and adults with recessive dystrophic epidermolysis bullosa, or RDEB, one of the most severe forms of the disease.^[13][16]

Clinical research continues to explore additional gene therapy approaches. Some studies are investigating whether delivering corrected genes through bone marrow transplants could produce lasting improvements. Others are examining whether gene editing technologies might permanently correct the mutations in a patient’s own cells. These approaches are currently in earlier phases of testing, with Phase I trials primarily assessing safety and appropriate dosing, while Phase II trials focus on determining whether the treatments actually improve symptoms and how well they work compared to existing therapies.^[15]

Researchers are also studying medications that might improve healing or reduce symptoms without directly correcting the genetic defect. Clinical trials have investigated calcipotriol, a topical medication related to vitamin D, to see if it can enhance wound healing in people with epidermolysis bullosa simplex. Another compound called diacerein, which reduces inflammation, is being tested as a topical treatment to help wounds heal faster. These trials typically take place at specialized EB treatment centers in the United States, Europe, and other regions around the world.^[15]

For people with severe EB who develop aggressive skin cancer that has spread or does not respond to standard treatments, researchers are investigating immunotherapy. This approach uses medications called monoclonal antibodies that help the body’s immune system recognize and attack cancer cells. One such medication being studied is an anti-PD1 antibody, which blocks a protein that cancer cells use to hide from the immune system. These trials are typically Phase II or Phase III studies that compare the new treatment to standard care or placebo treatments to determine effectiveness.^[15]

Clinical trials follow a structured progression through different phases. Phase I studies involve small numbers of participants and focus primarily on safety, determining what doses can be used without causing unacceptable side effects. Phase II trials enroll more people and assess whether the treatment actually works—does it help wounds heal faster, reduce blistering, or improve quality of life? These studies also gather more information about side effects and begin to compare the new treatment to existing options. Phase III trials are large studies that definitively compare the new treatment to current standard care, often through randomized assignment where some patients receive the new treatment and others receive standard care. Finally, Phase IV studies continue to monitor approved treatments after they reach the market to detect rare side effects or long-term benefits that might not have been apparent in earlier trials.^[15]

Early results from various clinical trials have shown promise. Gene therapy approaches have demonstrated the ability to produce functional collagen in treated areas, leading to improved wound healing and reduced blistering in some patients. The birch bark extract trials showed statistically significant improvements in healing time for partial thickness wounds. Immunotherapy trials for EB-related skin cancer have shown that some patients experience tumor shrinkage or disease stabilization. However, researchers emphasize that more long-term data is needed to fully understand the benefits and potential risks of these newer therapies.^[14][15]

Eligibility for clinical trials varies depending on the specific study. Some trials accept only patients with particular types or severities of EB, while others may have age restrictions. Researchers typically require documentation of genetic testing results confirming the EB diagnosis and the specific mutation involved. Patients must generally be healthy enough to participate safely, without active infections or other conditions that might complicate the study. Trial locations are often centered at major research hospitals and specialized EB treatment centers, though some studies may be conducted at multiple sites to make participation more accessible to families.^[13]

Most common treatment methods

• Wound care and skin protection
  • Carefully puncturing new blisters with sterile needles to prevent expansion while leaving the skin covering intact as natural protection
  • Applying non-stick dressings that protect wounds without adhering to fragile skin, changed regularly to maintain cleanliness
  • Using petroleum-based ointments and antibiotic creams to keep skin moist and prevent infection
  • Avoiding adhesive bandages and regular tape that tear skin upon removal
  • Protecting skin from friction through careful clothing selection, proper shoes, and activity modifications
• Topical medications
  • Filsuvez (birch triterpenes) topical gel approved for treating wounds in patients 6 months and older with junctional and dystrophic EB
  • Investigational topical treatments including calcipotriol and diacerein being studied in clinical trials for wound healing enhancement
• Gene therapy
  • Beremagene geperpavec (Vyjuvek), the first FDA-approved topical gene therapy that delivers functional COL7A1 genes to skin cells
  • Prademagene zamikeracel (Zevaskyn), an autologous cell sheet-based gene therapy using the patient’s own corrected skin cells
  • Experimental bone marrow transplant approaches and gene editing technologies under investigation in early phase trials
• Pain and symptom management
  • Medications to control pain from blisters and wounds, ranging from over-the-counter to prescription pain relievers
  • Anti-itch medications to reduce scratching that can cause new blisters
  • Oral antibiotics for treating widespread skin infections when present
• Nutritional support
  • Dietary modifications including soft foods and nutritional supplements when mouth or throat blistering makes eating difficult
  • Surgical placement of feeding tubes (gastrostomy tubes) for direct stomach feeding when oral intake becomes inadequate
  • Vitamin and mineral supplements to support wound healing and overall health
• Surgical interventions
  • Esophageal dilation procedures to widen the food passage when scarring causes narrowing
  • Surgical separation of fused fingers or toes caused by repeated blistering and scarring
  • Removal of suspicious skin lesions due to increased skin cancer risk in certain EB types
  • Reconstructive surgery to address scarring and deformities that limit function
• Immunotherapy for cancer
  • Anti-PD1 monoclonal antibodies being investigated for treating aggressive squamous cell carcinoma in EB patients
  • Used as palliative treatment for locally advanced or metastatic skin cancer unresponsive to previous therapies
• Multidisciplinary supportive care
  • Coordination between dermatologists, nutritionists, physical therapists, dentists, and specialized nurses
  • Physical therapy to maintain movement and prevent joint contractures from scarring
  • Dental care addressing tooth decay and other oral complications common in EB
  • Social work support for insurance navigation and connection to community resources