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Mashup Score: 1Rare Disease Report® | Medical News & Insights | HCPLive - 2 month(s) ago
The rare disease report® condition center is a comprehensive resource for clinical news and expert insights on rare disease report®. Read more at HCPLive.
Source: www.hcplive.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0Ricky Safer: What Clinicians Need to Know About PSC - 2 month(s) ago
Safer discusses how symptoms and quality of life often go unaddressed in the few clinician-patient interactions about primary sclerosing cholangitis.
Source: www.hcplive.comCategories: General Medicine News, Rare DiseaseTweet-
In this interview: Safer, a patient with primary sclerosing cholangitis, discusses how symptoms and quality of life often go unaddressed in clinician-patient interactions. https://t.co/84pn11rznI #RareDiseaseDay #RareDiseaseDay2024
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Mashup Score: 1
Phase 3 data presented at ASH 2023 show the gene therapy significantly reduced annualized bleeding rates and the number of total bleeds.
Source: www.hcplive.comCategories: General Medicine News, Rare DiseaseTweet-
From #ASH2023: Phase 3 data show etranacogene dezaparvovec (EtranaDez), a gene therapy from Hemgenix, significantly reduced annualized bleeding rates and the number of total bleeds in hemophilia B. https://t.co/9iYfjFraRf #RareDiseaseDay #RareDiseaseDay2024 @CGT_Live
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Mashup Score: 4FDA Approves Nirogacestat Tablets for Rare Desmoid Tumors - 5 month(s) ago
The approval is the first for a systemic therapy to treat desmoid tumors, offering patients an alternative to surgery.
Source: www.hcplive.comCategories: General Medicine News, Rare DiseaseTweet-
A #RareDisease win last night: The FDA has approved nirogacestat tablets (Ogsiveo) to treat progressing, painful desmoid tumors. Learn more about the systemic drug here: https://t.co/CfqqLrj1ht @HCPLiveNews $SWTX
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Mashup Score: 4FDA Approves Nirogacestat Tablets for Rare Desmoid Tumors - 5 month(s) ago
The approval is the first for a systemic therapy to treat desmoid tumors, offering patients an alternative to surgery.
Source: www.hcplive.comCategories: General Medicine News, Rare DiseaseTweet-
A #RareDisease win last night: The FDA has approved nirogacestat tablets (Ogsiveo) to treat progressing, painful desmoid tumors. Learn more about the systemic drug here: https://t.co/CfqqLrj1ht @HCPLiveNews $SWTX
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Mashup Score: 1Reviewing 2022 Advances in Childhood Interstitial Lung Disease and Non-CF Bronchiectasis - 9 month(s) ago
A year of research emphasizes the significance of collaboration, standardized approaches, and targeted therapies in rare pulmonary diseases.
Source: www.hcplive.comCategories: General Medicine News, Latest HeadlinesTweet-
A year of research emphasizes the significance of collaboration, standardized approaches, and targeted therapies in rare pulmonary diseases. https://t.co/pr7kHrywPa
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A year of research emphasizes the significance of collaboration, standardized approaches, and targeted therapies in rare pulmonary diseases. https://t.co/ZkT0HnhoVY
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Mashup Score: 0Lynn Malec, MD: Efanesoctocog alfa Reduces Bleeds, Treatment Burden for Hemophilia - 10 month(s) ago
Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug’s potential to address unmet needs within the hemophilia community.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug's potential to address unmet needs within the hemophilia community. https://t.co/SssxhSxHrq #ISTH2023 @isth #raredisease
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Mashup Score: 0Guy Young, MD: Prophylactic or Gene Therapy, and Shared Decision Making in Hemophilia - 10 month(s) ago
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.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
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 @GiulianaGrossi https://t.co/srV5u1Fj5G @GuyYoungMD @ChildrensLA #hemophilia #genetherapy #prophylaxis
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Mashup Score: 1
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.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
The American Indian/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://t.co/nOKRfpQymW #FetalMedicine #Womenshealth
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Mashup Score: 0Ambroxol Repurposing Improves Biochemical Markers in Gaucher Disease - 10 month(s) ago
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.
Source: HCP LiveCategories: Latest Headlines, Rare DiseaseTweet-
Promising findings in Gaucher disease treatment: Ambroxol demonstrates positive responses, improving hemoglobin levels, platelet counts, and biomarkers. A step towards affordable care options. https://t.co/pWXKV2vvnY
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That is it from us for #RareDiseaseDay2024! Thank you to all those who acknowledged the day and an even bigger thank you to all those pushing the field into the future! https://t.co/D2UPzWsf8w