Providing breaking news, patient stories and FDA updates, specific to those within the rare disease
A medical communication company that provides timely news, including FDA updates, patient stories, and clinical trial notifications to those interested in and actively involved in the rare diseases community.
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.
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
: 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
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
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.
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.
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.
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.
"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...
FDA approves Roctavian, the first gene therapy for adults with Hemophilia A.
Hear thoughts from the experts:
https://ow.ly/xR8050P1VoB
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.
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.
The has approved pegunigalsidase alfa for the treatment of adults with disease.
Read more about the new ERT agent and its clinical data here:
The FDA approval comes with supporting data showing the ERT's non-inferiority to agalsidase beta in controlled eGFR decline.
Today's FDA approval of sodium oxybate Lumryz marks the first once-at-bedtime oxybate treatment for individuals living with narcolepsy.
This marks the first and only FDA-approved once-at-bedtime oxybate for individuals living with narcolepsy.
Quixartinib treatment for AML is being reviewed for another 3 months so the
U.S. Food and Drug Administration
can assess updates to the proposed Risk Evaluation and Mitigation Strategies included in the NDA.
http://ow.ly/ouT050NPoSk
Innovative strategies to increase treatment adherence may be necessary to improve clinical outcomes for children with sickle cell anemia http://ow.ly/uQo550NOqNS
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...
People with sickle cell disease have higher infertility rates but report lower treatment-seeking behavior according to new data.
Individuals with sickle cell disease report a higher rate of infertility than the general population, but seeking fertility care treatment is lower.
Hemophilia is a rare bleeding disorder that affects millions worldwide. Today, on Hemophilia Awareness Day, let's raise awareness for the challenges of this .
Investigators address 2 questions: How can we better understand the biology of these rare disorders? How can we stimulate research and optimize the regulatory process to improve diagnosis and targeted treatment?
Many patients with sickle cell disease rely on Medicaid and face multifaceted barriers to treatment that are medical and nonmedical. Hear more from Dr. Rafeek Yusuf, director of Health Services Research at Be The Match:
http://ow.ly/yeyw50N8Icf
Medicaid enrollees with sickle cell disease have limited access to clinical trials and out-of-state benefits. Barriers to treatment are multifaceted and occur at different levels of care.
A rare victory to end .
The FDA has approved omaveloxolone as the first treatment indicated for Friedreich's ataxia! Read more here: http://ow.ly/Tvsy50N5qgm
The approval for omaveloxolone was supported by open-label data showing benefit of care for up to 3 years.
Exciting news for severe hemophilia A patients! Once-weekly efanesoctocog alfa provides superior bleeding prevention & improvements in physical, pain, and joint health
http://ow.ly/Kxf350MT6c7
Once-weekly efanesoctocog alfa is a safe and effective treatment for patients with severe hemophilia A, providing better outcomes than current treatment options by significantly reducing bleeding rates and improving physical health, pain, and joint health.
According to older literature, most cases of involve the lungs, but Dr. Matthew Lander explains why there could be more to the rare disease.
http://ow.ly/3QoP50MPWgn
According to older literature, most cases of sarcoidosis involve the lungs, but Dr. Matthew Lander explains why there could be more to the rare disease.
"The testing modality that's changing how we view cardiac sarcoidosis a lot is PET scanning," Dr. Matt Lander, Allegheny Health Network, explains in an interview with Giuliana Grossi, HCPLive. "Specifically with a tracer called FDG, which sometimes allows us to find areas of inflammation in the heart."
http://ow.ly/rKlA50MOOIM
PET scanning, in particular, has provided advancement in the diagnosis process, which has offered a new perspective on cardiac sarcoidosis for clinicians.
Learn about Cayenne Wellness Center and Children's Foundation's exciting new Sickle Cell Trait Awareness Campaign ( ) from leaders, Dr. Carolyn Rowley and Pat Corley, RN:
http://ow.ly/b5os50MLce6
The Sickle Cell Trait Awareness Campaign (STAC) offers promising possibilities of expanding sickle cell trait education across the country, Dr. Carolyn Rowley and Pat Corley, RN explain.
Currently, there are no FDA-approved therapies for , a rare autoantibody-driven disease that attacks fetal red blood cells during pregnancy potentially leading to fatal anemia.
Janssen Global
http://ow.ly/B17L50MKPQv
The rare condition can lead to life-threatening anemia in the fetus and currently lacks FDA-approved treatments.
The last week of each month, our editorial team compiles a list of our most popular rheumatology content from the past month for a month in review recap. The January 2023 rheumatology month in review highlights a recent study that observed an increased risk of hospitalization and death among patients with gout and COVID-19, the expansion of research evaluating adalimumab biosimilars, and trends in fibromyalgia.
http://ow.ly/EAs950MJsre
The January 2023 rheumatology month in review highlights a recent study that indicated an increased risk of hospitalization and death among patients with gout and COVID-19, the expansion of research evaluating adalimumab biosimilars, and trends in fibromyalgia.
Attacks from can be debilitating and even potentially fatal for pediatric patients. Lanadelumab (TAKHZYRO) is now available as a prophylaxis treatment for children aged 2 to
Lanadelumab (Takhzyro) has been approved for the treatment of hereditary angioedema in patients 12 and older.
No risk was identified between Guillain-Barré syndrome and mRNA vaccines BNT162b2 (Pfizer-BioNTech) and mRNA-1273 (Moderna) post-vaccination.
http://ow.ly/YTgC50MJrHU
The mRNA vaccines BNT162b2 (Pfizer-BioNTech) and mRNA-1273 (Moderna) showed no risk of Guillain-Barré syndrome post-vaccination.
Breaking news for Myelodysplastic Syndromes (MDS): Phase 3 clinical trial met primary and secondary endpoints with imetelstat, a novel telomerase inhibitor developed for hematologic malignancies.
http://ow.ly/YcUy50MiCxr
Promising results from the IMerge phase 3 clinical trial met the primary and secondary endpoints in the investigation of imetelstat, a novel telomerase inhibitor developed for hematologic malignancies.
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Dr. Mikkael Sekeres discusses the approval of quizartinib (Vanflyta) and how it impacts the treatment of acute myeloid leukemia #AML https://ow.ly/6kxP50Pqmi1 Sylvester Comprehensive Cancer Center
FDA approves Roctavian, the first gene therapy for adults with Hemophilia A. Hear thoughts from the experts: https://ow.ly/xR8050P1VoB #genetherapy #raredisease
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