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Rett Syndrome Research Trust Announces Development of the ORCA Measure to Assess Communication Ability of Individuals with Ret ( TRUMBUL )

Rett Syndrome Research Trust Announces Development of the ORCA Measure to Assess Communication Ability of Individuals with Ret ( TRUMBUL )
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United statesUnited kingdomBryce reeveMonica coenraadsAllyson berentFoundation for angelman syndrome therapeuticsObserver reported communications ability measureDuke university centerRett syndrome researchHealth measurementRett syndrome research trustDuke universityReported communicationsRett syndromeChief executive officerAngelman syndrome

Insight From Professional Liability Practice Leader Raffi Kodikian Into Rett Syndrome

Insight From Professional Liability Practice Leader Raffi Kodikian Into Rett Syndrome
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United statesSan franciscoAndreas rettRaffi kodikianNational institute of neurologicalRett syndrome researchRett syndromeNeurological disorder that mostly strikes femalesVice presidentLawyers professional liability practice leaderFounders professionalNational instituteNeurological ordersBengt hagbergKodikian applauds new work towardsRett syndrome research trust

Rett Syndrome Research Trust Establishes Translational Medicine Advisory Board

Rett Syndrome Research Trust Establishes Translational Medicine Advisory Board Share Article Four nationally prominent leaders in medical research join the board to advance progress toward a cure for Rett Syndrome “It is an honor to work with such distinguished colleagues on this advisory board; it is also a productive and very meaningful endeavor because of the intense commitment and unusually strong scientific sophistication of the Rett Syndrome Research Trust’s leadership,” said Dr. Hyman. TRUMBULL, Conn. (PRWEB) May 20, 2021 The Rett Syndrome Research Trust has established a Translational Medicine Advisory Board to provide new strategic direction for its CURE 360 agenda and open new avenues of innovative research that can be applied to Rett Syndrome. Four internationally recognized leaders in the field of medical research have joined the advisory board, all of whom have significant, broad academic and

United statesMonica coenraadsPhilip reillyStevene hymanPaul wangPaul nioiClinical development at seaside therapeuticsCore institute memberHarvard university provostClinical development at pfizerHead of medical research at autism speaksBoston university school of public healthStanley centerNational institute of mental healthBroad institute of harvardTranslational research at alnylam pharmaceuticals

Study finds antisense oligonucleotides to treat MECP2 disorder

Study finds antisense oligonucleotides to treat MECP2 disorder
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United statesBaylor college of medicineAlexanderj trostleSameers bajikarQi wangFrank rigoHuda zoghbiYehezkel sztainbergPaymaan jafar nejadZhandong liuJianrong tangYingyao shaoRegeneron pharmaceuticalsChao family foundationHamill foundationNational institutes of health

Antisense oligonucleotides as a feasible therapy to treat MECP2 duplication disorder

E-Mail Many cognitive neurodevelopmental disorders are a result of too many or too few copies of certain genes or chromosomes. To date, no treatment options exist for this class of disorders. MECP2 duplication syndrome (MDS) is one such disorder that primarily affects boys and results from a duplication spanning the methyl-CpG binding protein 2 (MECP2) locus located on the X chromosome. A preclinical study published from the laboratory of Dr. Huda Zoghbi, professor at Baylor College of Medicine and director of the Jan and Dan Duncan Neurological Research Institute at Texas Children s Hospital, provides experimental evidence that supports the use of antisense oligonucleotides as a feasible strategy to treat MDS. The study also offers crucial insights into the pharmacodynamics of this approach, which will serve as an important guide for the design and implementation of future clinical trials for this disorder. The study appears in the journal

United statesBaylor college of medicineAlexanderj trostleSameers bajikarQi wangFrank rigoHuda zoghbiYehezkel sztainbergPaymaan jafar nejadZhandong liuJianrong tangYingyao shaoRegeneron pharmaceuticalsChao family foundationHamill foundationNational institutes of health