On December 6, 2018, MASAC—the NHF’s Medical and Scientific Advisory Council—published recommendation #254, entitled, “MASAC Document Regarding Risks of Gene Therapy Trials for Hemophilia”.
MASAC’s recommendation offers a comprehensive view of many aspects to consider in gene therapy trials. It supports human clinical trials with gene therapy while emphasizing the importance of identifying and mitigating risks to patients, of addressing the unknown risks during the clinical trial process and of continued and open communication of all progress to the hemophilia community.
The Medical Advisory Board of the WFH supports MASAC’s, “MASAC Document Regarding Risks of Gene Therapy Trials for Hemophilia” published on December 6, 2018.
Click here to read the recommendation published by MASAC.
To read the WFH’s comments on the “Draft Guidance for Industry: Human Gene Therapy for Hemophilia”, sent to the U.S.A Food and Drug Administration (FDA), please click here.