Medical decisions in hemophilia care are primarily related to the type of factor replacement
and treatment regimen. With the growing number of treatment options for patients with …

Introduction National Member Organisations (NMO) of persons with haemophilia (PWH)
from the DACH Region (D= Germany, A= Austria, CH= Switzerland) were interested to better …

Introduction Exploring patient perceptions regarding gene therapies may provide insights
about their acceptability to patients. Objective To investigate opinions of people with …

Objective To examine patient/caregiver preference for key attributes of treatments for spinal
muscular atrophy (SMA). Background In the rapidly evolving SMA treatment landscape, it is …

Background: Hemophilia A is a chronic condition that requires meticulous treatment and
management. Patient preferences for prophylactic treatment can substantially influence …

Objectives To gain insights into the usage of factor VIII (FVIII) products by patients diagnosed
with moderate/severe hemophilia A, and to assess the impact and perceived importance of …

Introduction Reports on patients' satisfaction and preferred characteristics for treatments
would be worthwhile when choosing an optimal treatment reflecting patients' perspectives …

Introduction Prophylactic treatment of severe hemophilia A is burdensome, requiring
frequent intravenous injections. Extended half-life (EHL) factor VIII replacement therapies …

Introduction: Gene therapies are innovative therapies that are increasingly being developed.
However, health technology assessment (HTA) and payer decision making on these …

Background Current treatments for hemophilia A in Canada include on-demand treatment
as bleeds occur and regular intravenous prophylactic factor VIII (FVIII) infusions. The …