Rare Disease Report

28/11/2023

A win last night: The FDA has approved nirogacestat tablets (Ogsiveo) to treat progressing, painful desmoid tumors.

Learn more about the systemic drug here:

The approval is the first for a systemic therapy to treat desmoid tumors, offering patients an alternative to surgery.

01/08/2023

Dr. Mikkael Sekeres discusses the approval of quizartinib (Vanflyta) and how it impacts the treatment of acute myeloid leukemia
https://ow.ly/6kxP50Pqmi1 Sylvester Comprehensive Cancer Center

16/07/2023

: The latest feature in the Unreached Series
Experts share perspectives on how integrative care best serves their patients, practice, and the healthcare system overall.

Read more: https://ow.ly/psB950PcoB6

14/07/2023

The latest feature in The Unreached series delves into integrative care with expert perspectives from Baylor College of Medicine, Sickle Cell Disease Association of America, Inc., Texas Children's Cancer and Hematology Center, and more.

https://ow.ly/qum050Pc498

13/07/2023

Individuals seeking contraception will be able to access norgestrel without consulting a health care provider, reducing barriers to contraception access.
https://ow.ly/EbWc50PaEm3

Individuals seeking contraception will be able to access norgestrel without consulting a health care provider, reducing barriers to contraception access.

10/07/2023

Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug's potential to address unmet needs within the hemophilia community.
https://ow.ly/XkYq50P6YWw International Society on Thrombosis and Haemostasis (ISTH)

Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug's potential to address unmet needs within the hemophilia community.

08/07/2023

Psychological factors also come into play, as patients may have varying appetites for risk and preferences for treatment regimens, Dr. Guy Young explains in an interview with HCPLive
https://ow.ly/w4qC50P6Xl7 HEMLIBRA® (emicizumab-kxwh)

Informed discussions about the pros and cons of different treatments help guide the decision-making process. When it comes to treatment adherence and overall quality of life, patient lifestyle plays a significant role.

03/07/2023

The American Indian and Alaska Native population showed the largest increase in maternal mortality ratios rising from 14-49.2, while the Black population consistently had the highest median state ratios, increasing from 26.7-55.4
https://ow.ly/PoeM50P2O7m

The American Indian and Alaska Native population show the largest increase in median state ratios, rising from 14 - 49.2, while the Black population consistently had the highest median state ratios, increasing from 26.7 - 55.4.

02/07/2023

"Hemlibra is currently the only approved medicine for people with hemophilia A with or without factor VIII inhibitors that can be self-administered subcutaneously once weekly, every 2 weeks, or every 4 weeks," Dr. Juliana Biondo explains.
https://ow.ly/Cpoa50P28OM

HCPLive is a comprehensive clinical news and information portal that provides physicians with up-to-date specialty and disease-specific resources designed to help them provide better care to patients. At HCPLive, you will find breaking news, video interviews with physician experts, in-depth conferen...

30/06/2023

FDA approves Roctavian, the first gene therapy for adults with Hemophilia A.

Hear thoughts from the experts:
https://ow.ly/xR8050P1VoB

28/06/2023

Promising findings in Gaucher disease treatment: Ambroxol demonstrates positive responses, improving hemoglobin levels, platelet counts, and biomarkers. A step towards affordable care options.

Significant improvements seen in hematologic parameters, visceral volumes, and plasma biomarkers in patients with Gaucher disease, suggesting its potential as an alternative therapy, particularly for those who cannot access enzyme replacement therapy.

13/06/2023

Breaking news this afternoon from HCPLive: Odevixibat has been approved for pediatric patients with syndrome!

Hear from experts, including Dr. Nadia Ovchinsky of NYU Langone Health and Dr. Kris Kowdley of Washington State University on what this means for the rare liver disease:

The approval makes odevixibat the second IBAT inhibitor approved for the rare genetic disease, with capability to benefit both liver damage and itch symptoms.