News

December 16, 2020December 16, 2020

FLT180a Gene Therapy Can Prevent Bleeds in Hemophilia B Patients for Up to 3 Years, Data Show

News

A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX) within a normal range for nearly three years, effectively preventing ... Read more

December 14, 2020December 14, 2020

Dosing of Fitusiran in U.S. Hemophilia Trials to Resume

News

Dosing has been resumed in ongoing clinical trials in the U.S. testing the investigational medication fitusiran in adults and adolescents with hemophilia, Sanofi announced in a press release. The ... Read more

December 11, 2020December 11, 2020

High Dose of Gene Therapy SB-525 Raises FVIII Levels, Prevents Bleeds, Alta Trial Data Show

News

When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, ... Read more

December 9, 2020December 9, 2020

UniQure Shares Long-term Data About AMT-061 and AMT-060 Gene Therapy Candidates

News

Two potential gene therapies — AMT-060 and AMT-061 (etranacogene dezaparvovec) — lead to safe and long-term increases in factor IX (FIX) activity, as well as fewer bleeds among patients ... Read more

December 7, 2020December 7, 2020

MarzAA on FDA Fast Track to Treat Hemophilia A or B With Inhibitors

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment ... Read more

December 4, 2020December 4, 2020

myPKFiT Software for Adynovate Now Available in the US

News

A web-based software to help personalize dosing regimens for hemophilia A patients being treated with Adynovate is now available in the U.S., according to Takeda. The software, called myPKFiT, previously ... Read more

December 2, 2020December 2, 2020

Gene Therapy AMT-060 for Hemophilia B Prevents Bleeds Up to 5 Years, Ongoing Trial Shows

News

AMT-060, a first-generation gene therapy candidate by uniQure, continues to safely reduce the number of bleeding episodes and the use of clotting factor IX (FIX) in men with moderate ... Read more

December 1, 2020December 1, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders ... Read more

November 30, 2020November 30, 2020

Most Hemophilia Patients Show Positive Attitude for Gene Therapy in Study

News

Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a small interview study assessing patient ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more