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Mashup Score: 1Nipocalimab Benefits Mechanism of HDFN in Phase 2 Trial - 11 month(s) ago
Janssen reported significant benefit, and a tolerable profile, for the investigative drug that may become the first indicated to treat HDFN.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 0Nadia Ovchinsky, MD: Odevixibat's Phase 3 Data and Potential for Alagille Syndrome - 11 month(s) ago
The IBAT inhibitor will receive an FDA decision by next week. Ovchinsky reviews its supporting data for benefitting patient pruritus, sleep, and liver bile acid levels.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
As we near the FDA decision date, #odevixibat phase 3 study author @Dr_NOvchinsky shares her opinion on how the IBAT inhibitor may benefit real-world children with the rare Alagille syndrome: https://t.co/xpZHzjyAz9 https://t.co/umVRp80EHc
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Mashup Score: 3
Qualitative findings show children with sickle cell disease have the desire to feel normal and utilize personal strengths to manage their condition.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
Hospitalization for sickle cell disease is described as overwhelming and disruptive to everyday life, motivating young patients to take control and seek effective pain management strategies. https://t.co/iSK0FkFnzX
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Mashup Score: 0
Results show a high level of adherence to hydroxyurea medication is essential for lowering the incidence of negative clinical outcomes like VOCs and acute chest syndrome in children with sickle cell anemia.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
Innovative strategies to increase treatment adherence may be necessary to improve clinical outcomes for children with sickle cell anemia https://t.co/izfSfNQNHR #hydroxyurea #SCD #Pediatric
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Mashup Score: 0
Individuals with sickle cell disease report a higher rate of infertility than the general population, but seeking fertility care treatment is lower.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
People with sickle cell disease have higher infertility rates but report lower treatment-seeking behavior according to new data. https://t.co/G24VsgZOM8
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Mashup Score: 0
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?
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
Hemophilia is a rare bleeding disorder that affects millions worldwide. Today, on Hemophilia Awareness Day, let's raise awareness for the challenges of this #RareDisease. #HemophiliaAwarenessDay https://t.co/Y3mSPmQa9J
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Mashup Score: 1
Phase 2/3 data show significant improvement in lymphoproliferation as measured by the reduction in lymph node size and increase in naïve B cells in patients with activated phosphoinositide 3-kinase delta syndrome.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
FDA approves leniolisib making it the first approved therapy for activated phosphoinositide 3-kinase delta syndrome #APDS #joenja @US_FDA https://t.co/5aA3OWz8J5
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Mashup Score: 0
While this population was previously thought to be unaffected, studies within the last few years have shown that women with hemophilia experience joint and bleeding problems.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
While this population was previously thought to be unaffected, studies within the last few years have shown that women with hemophilia experience joint and bleeding problems. #HemophiliaA #ALTUVIIIO https://t.co/w3qJuQrS8B
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Mashup Score: 0
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.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
Referring physicians' willingness to accept Medicaid insurance impacts the overall care of patients' with sickle cell disease. https://t.co/h08XxwDKgy #RareDiseaseDay2023 #SickleCell @BeTheMatch
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Mashup Score: 0
Dr. Margaret Ragni discusses the FDA approved, first-in-class, factor VIII replacement therapy, antihemophilic factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl, previously known as efanesoctocog alfa.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
"It's a much reduced burden of treatment with once-a-week intravenous infusion that gets you to a higher level the entire week, above about 15% so that you rarely have breakthrough bleeds" Dr. Margaret Ragni explains in an interview with @GiulianaGrossi https://t.co/ppgrmtUcmu
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New phase 2 data support the development of nipocalimab for the treatment of #HDFN. Read about Janssen's announcement of the data from earlier this morning—and what it may mean for the hemolytic disease: https://t.co/y3zyvUeBu9 #RareDisease #FDA