FDA Expands ALS Oral Drug Riluzole to Feeding Tube Use
The new indication will allow the longtime ALS therapy to be used among the 85% of patients required to use PEG due to dysphagia.
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Rare Disease Report® | Medical News & Insights | HCPLive
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Ricky Safer: What Clinicians Need to Know About PSC
Safer discusses how symptoms and quality of life often go unaddressed in the few clinician-patient interactions about primary sclerosing cholangitis.
Etranacogene Dezaparvovec Significantly Improves Hemophilia B Past 2 Years
Phase 3 data presented at ASH 2023 show the gene therapy significantly reduced annualized bleeding rates and the number of total bleeds.
FDA Approves Nirogacestat Tablets for Rare Desmoid Tumors
The approval is the first for a systemic therapy to treat desmoid tumors, offering patients an alternative to surgery.
FDA Approves Nirogacestat Tablets for Rare Desmoid Tumors
The approval is the first for a systemic therapy to treat desmoid tumors, offering patients an alternative to surgery.
Reviewing 2022 Advances in Childhood Interstitial Lung Disease and Non-CF Bronchiectasis
A year of research emphasizes the significance of collaboration, standardized approaches, and targeted therapies in rare pulmonary diseases.
Lynn Malec, MD: Efanesoctocog alfa Reduces Bleeds, Treatment Burden for Hemophilia
Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug’s potential to address unmet needs within the hemophilia community.
Guy Young, MD: Prophylactic or Gene Therapy, and Shared Decision Making in Hemophilia
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…
Maternal Mortality Rates Highlight Alarming Inequities by State, Race/Ethnicity
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…
Ambroxol Repurposing Improves Biochemical Markers in Gaucher Disease
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…