Informational Videos


Dyskeratosis Congenita-specific Bone Marrow Transplantation: Less Toxicity, Better Outcomes

Dr. Jakub Tolar, M.D., Ph.D.
Director, Stem Cell Institute, University of Minnesota
Associate Professor, Pediatrics, Blood and Marrow Transplantation
Attending Physician, Amplatz Children's Hospital, University of Minnesota, Fairview

BMT for DC: Late Effects and New Approaches

Dr. Suneet Agarwal, M.D., Ph.D. 
Assistant Professor, Harvard Medical School
Staff Physician, Stem Cell Trasplantation, Dana-Farber/Boston Children's Cancer and Blood Disorders Center

Dyskeratosis Congenita Management and Treatment

Dr. Alison Bertuch, M.D., Ph.D. 
Associate Professor of Pediatrics and Molecular and Human Genetics, Baylor College of Medicine
Director, Bone Marrow Failure Program, Texas Children's Cancer and Hematolgy Centers

Pulmonary Fibrosis in Dyskeratosis Congenita

Dr. Ivan O. Rosas, M.D.
Assistant Professor of Medicine, Harvard Medical School
Director, Interstitial Lung Disease Program, Brigham and Women's Hospital


Rare Disease; The Path Less Chosen

Part of a continuing series of videotaped sessions at Boston Children’s Hospital’s recent Global Pediatric Innovation Summit + Awards 2014.

Rare diseases offer a lot of opportunity for gene discovery, but getting a drug to market presents many challenges, and costs per patient are high. This 50-minute session explored this complicated landscape from multiple angles. The panelists:



The ZKRD - the German National Registry of Blood Stem Cell Donors is the nation’s information hub in searching for an unrelated blood stem cell donor. Data from all over Germany pertaining to the search for an unrelated donor converges here.Therefore, the actual search for a suitable donor is processed here.

As a centre of competence for this highly specialised field of medical care, the ZKRD organises the search for donors at a national and international level in order to assure that this complex process operates as quickly, efficiently, transparently and economically as possible. It coordinates the search process and checks all related requests and results for completeness and plausibility.

Its service framework includes coordinating all steps from the initial request to invoicing the cost units. Additionally, the ZKRD fulfils a wide array of consulting and organisational functions.

Along with the U.S. National Marrow Donor Program (NMDP), the ZKRD holds a leading position at an international level. With about 5.5 million donors, it is the second largest registry. If ranking is based on the number of donors identified annually for transplantation, then the ZKRD is one of the most productive registries worldwide.


Travel Resources

Miracle Flights Charity Pairs Rare Patients With Essential Doctors

To qualify for Miracle Flights' assistance to fly to non-research medical appointments, families must provide a letter from both a referring and an accepting physician and have a low to moderate income. This program is for children less than 18 years of age. They are allowed to be accompanied by two parents or the child's eligible caregiver. The completed request must be made no more than 60-90 days and no later than 14 days before the appointment. International patients can also qualify to be flown into the U.S.  For more information, call 800-359-1711 or click the website link.

Financial Support

The Julia’s Wings Foundation

Julia's Wings Foundation is a non-profit organization, certified 501(c)(3), that provides financial assistance to families of children with the life threatening hematological diseases, aplastic anemia, MDS and PNH. The fund has a budget of $75,000 per calendar year for the purpose of providing  financial support to these families to cover costs of medicines not covered by insurance, travel, lodging, and other living expenses for qualified participants. If you are a parent or guardian of a child with aplastic anemia and have been forced to incur significant financial burdens in the process of getting your child the medical treatment they require, you may be eligible to receive financial assistance from the Julia’s Wings Foundation. We offer short-term financial assistance to families of children with aplastic anemia, particularly those in the midst of treatment or prolonged hospitalizations. See above  link for more information and click here for the application process.