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'Bubble kid' success puts gene therapy back on track
RESEARCH Mar 19, 2015 | 23:25 pm
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MOST parents dream of a 5-week-old baby who sleeps through the night, but Aga Warnell knew something was wrong. Her baby, Nina, just wasn't hungry in the way her other daughters had been.Within weeks, Nina became very ill, says her[…] -
Gene Therapy Research
RESEARCH Jan 11, 2015 | 13:13 pm
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Gene therapy emerged 15 yr ago with great expectations for a marriage between the remarkable advances in molecular biology of the previous decade and clinical medicine. It was originally an innovative treatment for incurable diseases, so-called genetic disorders, in which[…] -
Bartter Syndrome and Gitelman Syndrome
RESEARCH Dec 28, 2014 | 17:21 pm
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Bartter syndrome and Gitelman syndrome are characterized by fluid, electrolyte, urinary, and hormonal abnormalities, including renal K, Na, Cl, and H wasting; hypokalemia; hyperreninemia and hyperaldosteronism without hypertension; and metabolic alkalosis. Findings include electrolyte, growth, and sometimes neuromuscular abnormalities. Diagnosis[…] -
The Salty Six: Six Foods That Load Your Diet With Salt
NUTRITION Dec 28, 2014 | 17:05 pm
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Eating too many salty foods can create all sorts of health problems, including high blood pressure. What you may not know is that a lot of common foods are packed with excess sodium.“Excess sodium in our diets has less to[…]
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Rare Disease Day 2018
NEWS Jan 5, 2018 | 07:41 am
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There has been great progress in rare disease research, in big part thanks to the advocacy work of the rare disease patient community. Patients are already participating in research. And in some cases patients have taken the reins themselves to fund their own research.However, the fact remains that there are[…] -
Differences Between Bartter & Gitelman Syndrome
ETHAN'S JOURNEY Feb 1, 2017 | 04:16 am
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Kasich Signs Six Bills
NEWS Dec 19, 2016 | 22:18 pm
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COLUMBUS – Today Governor John R. Kasich signed the following bills into law: H.B. 166 (Green) extends the deadline for filing an application for the homestead exemption or 2 1/2% property tax rollback to the end of the tax year, to require that auditors certify Local Government Fund allocations to[…] -
Ohio Rare Disease Day 2016
ETHAN'S JOURNEY Feb 17, 2016 | 15:25 pm
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Created by Ethan Hoffman
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Senate Approves Bartter Syndrome Legislation From Senators Schiavoni And Cafaro
EVENTS Dec 19, 2016 | 22:08 pm
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COLUMBUS - Yesterday, Senator Joe Schiavoni (D-Boardman) and Senator Capri S. Cafaro (D-Hubbard) introduced S.B. 128, which would designate May 30th as Bartter Syndrome Awareness Day. The Senate passed S.B. 128 with unanimous support.“Bartter Syndrome is not well known, which often means those suffering from it wait far too long[…] -
Congressmen Tim Ryan and David Joyce Introduce Resolution to Designate National Bartter Syndrome Day
EVENTS Dec 19, 2016 | 22:03 pm
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Washington, D.C. – Congressman Tim Ryan (D-OH) and Congressman David Joyce (R-OH) today introduced a resolution to designate each May 30th as National Bartter Syndrome Day. This designation would give much needed attention and increase awareness for the one million people worldwide affected by Bartter Syndrome and their families working[…]
In the News
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Gene therapy drugs step toward approval in JapanPosted on: 14 November 2018, source: Nikkei Asia
Novartis has...- Infectivity of adeno-associated virus serotypes in mouse testis
Posted on: 7 November 2018, source: BMC Biotechnology
In a... - Infectivity of adeno-associated virus serotypes in mouse testis