Part 1: Rare Disease Clinical Research – Spotlight on the Patient and...
In this webinar, Medpace partners with the Batten Disease Support and Research Association (BDSRA) to discuss the need to educate patients and their caregivers on the importance and increase of...(PRWeb February 13, 2020)Read the full story at https://www.prweb.com/releases/part_1_rare_disease_clinical_research_spotlight_on_the_patient_and_caregiver_upcoming_webinar_hosted_by_xtalks/prweb16908279.htm
Pulmonary artery sarcoma (PAS) is an exceedingly rare disease with historically poor survival. PAS is often mistaken for chronic thromboembolic pulmonary hypertension (CTEPH) owing to similarities in clinical presentation. Controversy exists regarding need to definitively differentiate PAS from CTEPH prior to surgical therapy. Furthermore lung resection would seem inferior palliation to tumor endarterectomy in a predominantly bilateral disease dominated by hemodynamic compromise.
Following introduction of the lung allocation score (LAS) in 2011, Eurotransplant member centers can apply for an exceptional LAS (eLAS) if the calculated LAS insufficiently reflects the perceived transplant benefit for a patient, specifically in case of primary pulmonary hypertension group 1 and 4; combined lung+non ‐renal transplantation; rare diseases; or extracorporeal support. Each eLAS proposal is evaluated by a LAS Review Board, consisting of ≥3 lung transplant experts, which subsequently declines or approves the eLAS request in consensus of ≥3 votes.
Fulminant myocarditis (FM) is a rare disease that may progress rapidly to refractory cardiogenic shock requiring temporary mechanical circulatory support. Venoarterial extracorporeal membrane oxygenation (VA-ECMO) has been used to provide biventricular support in these patients, however, studies have demonstrated conflicting results, likely due to small sample size. We conducted a systematic review and meta-analysis to evaluate short-term mortality after VA-ECMO in patients with FM.
In conclusion, a JEM can be a very handy too l for exposure assessment in occupational epidemiology, particularly in large-scale studies with limited occupational information. When selecting the most suitable exposure assessment method, however, researchers should always remain critical. Know when a JEM has added value and recognize its limita tions. References 1. Hoar SK, Morrison AS, Cole P, Silverman DT. An occupation and exposure linkage system for the study of occupational carcinogenesis. J Occup Med. 1980;22(11):722-6. 2. Pannett B, Coggon D, Acheson ED. A job-exposure matrix for use in population based studies in ...
Findings provide insight that may inform search for treatments.
The objective of this paper is to answer some of the more frequent questions related to the management of patients with XLH by a group of experts participating in a scientific conference on XLH held in Madrid.
AbstractRare diseases are heterogeneous life-threatening or seriously debilitating conditions that affect
(Natural News) A rare disease called limb-girdle muscular dystrophy D1 (LMGD1) makes even everyday actions such as climbing stairs, lifting objects and standing up from a chair difficult to do. Over time, it can even cause some people to lose the ability to walk. A recent study, however, shows that lithium can be a possible treatment for LMGD1. Researchers...
Discussion section as follows.
Authors: Laforgia M, Calabrò C, Scattone A, Laface C, Porcelli M, Gadaleta CD, Nardulli P, Ranieri G Abstract Introduction: Mast cell leukemia (MCL) is one of the most aggressive forms of Systemic Mastocytosis (SM), a complex family of rare diseases, for which standard therapies are very few. MCL represents only