Replaces: 277INTRODUCTION Successful gene therapy will require broad-based, multi-stakeholder participation to define patient eligibility, educate health care providers, educate persons with hemophilia (PWH) and families, and…
Novo Nordisk has notified the National Hemophilia Foundation that they have received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for their investigational, subcutaneous therapy concizumab.
Concizumab …
Between 2013-2017, the “My Life Our Future” (MLOF) project offered eligible individuals with hemophilia free genotyping, which is historically hard to access, expensive, and not covered by insurance. Conducted through the laboratory analysis of a…
In July 2020 Ferring Pharmaceuticals suspended manufacturing of the nasal spray STIMATE®, a popular product used to treat certain bleeding disorders. The suspension, and subsequent pharmacy level recall, were in response to a packaging seal…
NHF’s Medical and Scientific Advisory Council (MASAC) recently issued four new documents, which are now available for easy access.Document 275 emphasizes the pivotal part physical therapy plays in the integrated…
We invite you to join the quarterly board of directors meeting for the National Hemophilia Foundation on May 18th starting at 7:00 pm ET. This meeting will discuss NHF business operations and decisions and is…
The Rare Diseases Diversity Coalition (RDDC) is currently seeking applicants for its 2023 Rare Disease Fellowship Program, which is aimed at inspiring the next generation of the medical workforce to work in the rare disease space and…