Fidest – Agenzia giornalistica/press agency

Quotidiano di informazione – Anno 33 n° 335

Posts Tagged ‘disorders’

FDA approves Roche’s Rituxan (rituximab) in children with two rare blood vessel disorders

Posted by fidest press agency su giovedì, 3 ottobre 2019

Basel Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the U.S. Food and Drug Administration (FDA) has approved Rituxan® (rituximab), in combination with glucocorticoids, for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in paediatric patients 2 years of age and older. GPA and MPA are rare, potentially life-threatening diseases affecting small and medium sized blood vessels.
The approval is based on data from the PePRS study, a Phase IIa, global, open-label, multicentre single-arm study investigating the safety, pharmacokinetics, exploratory efficacy and pharmacodynamic outcomes of intravenous MabThera/Rituxan in 25 patients with active GPA or MPA between 6 and 17 years of age. Treatment with four weekly infusions of MabThera/Rituxan in combination with a tapering course of oral glucocorticoids was assessed in newly diagnosed or relapsing active GPA or MPA paediatric patients. Of the 25 patients in the study, 19 had GPA and 6 had MPA at baseline. Efficacy was an exploratory endpoint and primarily assessed using the Paediatric Vasculitis Activity Score (PVAS). Efficacy assessment showed that 56% of patients achieved PVAS remission by month 6, 92% by month 12, and 100% of patients achieved remission by month 18. The safety profile of MabThera/Rituxan in patients with paediatric GPA and MPA was consistent in type, nature and severity with the known safety profile of MabThera/Rituxan in adult patients with GPA and MPA, rheumatoid arthritis and pemphigus vulgaris.The FDA previously granted Priority Review to Rituxan for the treatment of GPA and MPA in paediatric patients. In 2011, Rituxan became the first and only therapy approved by the FDA for the treatment of adults with these two rare forms of vasculitis. MabThera/Rituxan is currently indicated for the treatment of four autoimmune conditions and since 2006 more than 900,000 people have been treated with MabThera/Rituxan for autoimmune conditions worldwide. MabThera/Rituxan is not indicated in children less than 2 years of age with GPA or MPA, or in children with conditions outside of GPA and MPA.

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Two Rare and Devastating Neurodegenerative Disorders

Posted by fidest press agency su domenica, 10 febbraio 2019

Neurogene, Inc., a company with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, today announced that researchers presented preliminary, unpublished “proof of concept” data in mice evaluating the safety and efficacy of adeno-associated virus (AAV) gene therapy for AGU and CMT4J, two rare neurological diseases, at the 15th Annual WORLDSymposiumTM conference in Orlando, Florida.
The potential therapies are being investigated under a collaboration agreement between Neurogene and UT Southwestern Medical Center through multiple sponsored agreements. Neurogene provides financial support for these and other related research projects, including pre-clinical studies for AGU and CMT4J.AAV gene therapy is being investigated as a potential treatment for aspartylglucosaminuria (AGU), a neurodegenerative disease caused by dysfunction of the AGA gene. The unpublished data, which have not been peer reviewed, consider the safety and efficacy of gene therapy for these diseases in mice in support of potential future clinical trials. Xin Chen, Ph.D., Instructor of Pediatrics at UT Southwestern Medical Center, presented data evaluating the effects in mice lacking a functional AGA gene, using intravenous or intrathecal administration with low and high doses, and data were reported for early symptomatic mice. Dr. Chen’s team reported data indicating AGU gene therapy resulted in dose-dependent, complete or near-complete elimination of toxic substrate in central and peripheral tissues and body fluids and that treatment was well-tolerated at supraphysiological levels.

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Stressful jobs are associated with a higher risk of heart rhythm disorders

Posted by fidest press agency su martedì, 5 giugno 2018

Sophia Antipolis, 04 June 2018: Having a stressful job is associated with a higher risk of a heart rhythm disorder called atrial fibrillation, according to research published today in the European Journal of Preventive Cardiology, a European Society of Cardiology (ESC) journal.The most stressful jobs are psychologically demanding but give employees little control over the work situation – for example, assembly line workers, bus drivers, secretaries, and nurses. The study found that being stressed at work was associated with a 48% higher risk of atrial fibrillation, after adjustment for age, sex, and education. Dr Eleonor Fransson, study author and associate professor of epidemiology, School of Health and Welfare, Jönköping University, Sweden, said: “We need people to do these jobs but employers can help by making sure staff have the resources required to complete the assigned tasks. Bosses should schedule breaks and listen to employees’ ideas on how the work itself and the work environment can be improved.” Atrial fibrillation is the most common heart rhythm disorder (arrhythmia). Symptoms include palpitations, weakness, fatigue, feeling light headed, dizziness, and shortness of breath.
Atrial fibrillation causes 20–30% of all strokes and increases the risk of dying prematurely.2 One in four middle-aged adults in Europe and the US will develop atrial fibrillation. It is estimated that by 2030 there will be 14–17 million patients with atrial fibrillation in the European Union, with 120,000–215,000 new diagnoses each year.Dr Fransson said: “Atrial fibrillation is a common condition with serious consequences and therefore it is of major public health importance to find ways of preventing it. Little is known about risk factors for the disease and especially the role of the work environment.”This study assessed the link between work stress and atrial fibrillation. The study included 13,200 participants enrolled into the Swedish Longitudinal Occupational Survey of Health (SLOSH) in 2006, 2008, or 2010. Participants were employed and had no history of atrial fibrillation, heart attack, or heart failure. At study inclusion, participants completed postal surveys on sociodemographics, lifestyle, health, and work-related factors.Work stress was defined as job strain, which refers to jobs with high psychological demands combined with low control over the work situation. The survey included five questions on job demands and six on control – for example: Do you have to work very hard or very fast? Are there conflicting demands in your work? Do you have enough time to complete your work tasks? Does your work include a lot of repetition? Can you decide how and what to do at work? During a median follow-up of 5.7 years, 145 cases of atrial fibrillation were identified from national registers.
Dr Fransson said: “In the general working population in Sweden, employees with stressful jobs were almost 50% more likely to develop atrial fibrillation. The estimated risk remained even after we took into account other factors such as smoking, leisure time physical activity, body mass index, and hypertension.” The authors then pooled their results with two other studies on the same topic, and found that job strain was associated with a 37% increased risk of atrial fibrillation. “Across studies there was a consistent pattern of work stress being a risk factor for atrial fibrillation,” said Dr Fransson.She concluded: “Work stress has previously been linked with coronary heart disease. Work stress should be considered a modifiable risk factor for preventing atrial fibrillation and coronary heart disease. People who feel stressed at work and have palpitations or other symptoms of atrial fibrillation should see their doctor and speak to their employer about improving the situation at work.” European guidelines on the prevention of cardiovascular disease state that stress at work contributes to the risk of developing cardiovascular disease and having a worse prognosis.3 Assessment of psychosocial risk factors is recommended in people who have, or are at risk of developing, cardiovascular disease.

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