NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops…
Articles by Marisa Wexler, MS
Good Long-term Outcomes After Knee Replacement in Hemophilia Patients, Study Finds
Most people with hemophilia who undergo knee replacement surgery have favorable outcomes, including less pain and improved function,…
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Cancer Therapy Daratumumab Can Effectively Lower Inhibitors in Hemophilia A, Researchers Say
Daratumumab — a cancer therapy marketed as Darzalex — can reduce levels of factor VIII (FVIII) inhibitors in…
BIVV001 Safely Treats Severe Hemophilia A as Weekly Injection, Trial Reports
A single dose of BIVV001 is safe and effectively increases factor VIII (FVIII) activity over one month in men with…
Rebinyn/Refixia Effective and Safe in Untreated Boys With Hemophilia B, Data Suggest
Refixia or Rebinyn (nonacog beta pegol, or N9-GP), an approved replacement therapy for hemophilia B, is…
Early Data Support Catalyst Biosciences’ Potential Hemophilia B Gene Therapy
CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, can increase factor…
Roughly 30,000 Males With Hemophilia in the US, Study Estimates
Approximately 30,000 males have hemophilia in the U.S., according to estimates from a recent study, which also found that…
3 Hemophilia B Patients Report AMT-060 Gene Therapy Gave Them Normalcy
Three hemophilia B patients said they were able to lead a relatively normal life, including being more active and…
DalcA Effective and Safe as Hemophilia B Injection Treatment, Study Suggests
Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding…