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December 21, 2018December 20, 2018

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to ... Read more

December 14, 2018December 14, 2018

Mothers of Children with Rare Genetic Syndromes Experience Stress Similar to Moms of Autistic Kids, Study Finds

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A significant proportion of mothers of children with rare genetic syndromes, such as Angelman syndrome, experience levels of stress similar to those reported by mothers of children with autism ... Read more

December 10, 2018December 10, 2018

Ovid Therapeutics to Start Phase 3 Trial of OV101 in Children with Angelman Syndrome

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Ovid Therapeutics is planning to start a pivotal Phase 3 trial of OV101 (gaboxadol), its investigational oral therapy, in children ages 4-12 with Angelman syndrome. The company’s decision is based ... Read more

December 7, 2018December 7, 2018

Newer Anti-epileptic Therapies May Be Safer, More Effective in Reducing Seizures in Angelman’s Patients

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Newer anti-epileptic medications — such as Keppra (levetiracetam), Lamitcal (lamotrigine), and clobazam — as well as low glycemic index treatment may be more effective in reducing epileptic seizures and ... Read more

November 30, 2018November 30, 2018

‘World’s Largest’ Newborn Screening Study Includes Angelman Syndrome

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If successful, an extensive pilot screening study afoot in Australia for Angelman syndrome and three other rare genetic disorders could lead to large-scale testing. With a contribution from the ... Read more

November 16, 2018November 16, 2018

Short-term use of NSI-189 Aids Cognitive and Motor Skills in Angelman Mouse Model, Study Says

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Short-term treatment with Neuralstem‘s NSI-189 reversed cognitive and motor impairments in a mouse model of Angelman syndrome, according to study results. This early finding could help to pave the way for this compound, in ... Read more

November 9, 2018November 9, 2018

UBE3A Gene Reactivation in Inhibitory Neurons May Prevent Seizures, Angelman Mouse Study Shows

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Epileptic seizures caused by disturbances in the activity of a specific type of nerve cell called an inhibitory neuron were prevented by the reactivation of the UBE3A gene in young mice with Angelman ... Read more

November 2, 2018November 2, 2018

Researchers Closer to Understanding Role of Enzyme Behind Angelman Syndrome, Study Reports

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Researchers from the University of North Carolina have found that ubiquitin protein ligase E3A — the malfunction of which is behind Angelman syndrome development — is present in different types of neurons ... Read more

October 29, 2018October 29, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit ... Read more

October 26, 2018October 26, 2018

OV101 Improves Motor and Sleep Performance in Angelman Patients, New Data from STARS Phase 2 Trial Show

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New data from Ovid Therapeutics’ Phase 2 STARS trial shows that treatment with oral candidate OV101 once daily at a dose of 15 mg significantly improves sleep and motor skills ... Read more

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