Rare Disease News, Perspectives & Resources - Rare Disease Advisor
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2024年12月18日 · The yearly battle to gain insurance approvals tests the physical resilience and emotional strength of an individual with Pompe disease.
Spinal Muscular Atrophy Clinical Trials - Rare Disease Advisor
As of November 2024, approximately 137 clinical trials for spinal muscular atrophy (SMA) are ongoing or pending: 8 trials that are not yet recruiting, 74 trials that are currently recruiting, 26 trials that are active but no longer recruiting, 5 trials that are enrolling by invitation only, and 24 trials of unknown status. Of these 137 clinical trials, 67 are observational (including 14 ...
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Read about Rare Disease Advisor here. RareDiseaseAdvisor.com is the web’s premier source of news, perspectives, and resources for clinicians who treat patients with rare diseases.The website addresses the unmet informational needs of healthcare professionals seeking the latest insights into the rare diseases they treat through fresh, original, and authoritative content.
The 10 Biggest Stories in Rare Disease for 2023
2023年12月13日 · Dizziness is common among patients with MG, found a new Australian study published in the Journal of Clinical Neuroscience, “and warrants further investigation.”The study also found that awareness about the role of relevant allied health services and their utilization varies among patients.
Sickle Cell Disease - Rare Disease Advisor
Follow Rare Disease Advisor's Sickle Cell Disease Junction for the latest news, resources, insights, and patient perspectives on the disease.
AVO Therapy Effective and Well Tolerated in Patients With CLL
2 天之前 · Combination therapy with acalabrutinib, venetoclax, and obinutuzumab (AVO) is found to be highly active and well tolerated in treatment-naïve patients with chronic lymphocytic leukemia (CLL) and high-risk TP53 aberrations, according to results of a new phase 2 study published in the Journal of Clinical Oncology. “We previously reported early results of a phase II, investigator-initiated ...
ANCA-Associated Vasculitis Life Expectancy - Rare Disease Advisor
Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a heterogeneous group of rare autoimmune diseases in which ANCA target and activate neutrophils. These, in turn, attack the cells lining small blood vessels, causing inflammation and damage. 1 Depending on which organ or system is affected, the symptoms of AAV can vary greatly.
Epysqli - Rare Disease Advisor
2024年11月20日 · Epysqli® (eculizumab-aagh) is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. Epysqli is a biosimilar of Soliris® (eculizumab), which means there are no clinically meaningful differences between the 2 drugs in regard to safety, purity, and ...
Pompe Disease - Acid Maltase Deficiency | Rare Disease Advisor
Pompe disease, also known as acid maltase deficiency or glycogen storage disease type II, is a rare genetic disorder characterized by a deficiency or absence of the lysosomal acid alpha-glucosidase enzyme (GAA).GAA normally cleaves glycogen to form glucose. Abnormalities of the GAA enzyme due to mutations in the GAA gene result in an accumulation of glycogen in tissues, especially cardiac and ...