Clinical Trials Are in the Works That Will Test a Treatment for Phelan-McDermid Syndrome
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Clinical Trials Are in the Works That Will Test a Treatment for Phelan-McDermid Syndrome

According to a story from Newswise, the Seaver Autism Center for Research and Treatment at Mount Sinai recently announced that patient enrollment has begun for a clinical trial that will…

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By Observing the Effects of Alagille Syndrome, Researchers Discover How Liver Tissue Can Transform and Heal
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By Observing the Effects of Alagille Syndrome, Researchers Discover How Liver Tissue Can Transform and Heal

According to a story from PR Newswire, a group of scientists have managed to discover a mechanism that allows the liver to repair damage it has sustained. This discovery may…

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Most Epilepsy Patients Benefit From Current Medications, But Look Forward to New Treatments

According to a story from Scimex, a recent assessment of available treatments for Dravet syndrome and other types of epilepsy has reaffirmed that anticonvulsant medications are still the most effective…

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ICYMI: Experimental Drug for Spinal Muscular Atrophy Gets Orphan Drug Designation
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ICYMI: Experimental Drug for Spinal Muscular Atrophy Gets Orphan Drug Designation

According to a story from BioSpace, the pharmaceutical company Scholar Rock recently announced that its drug candidate SRK-015 was recently granted Orphan Drug Designation by the U.S. FDA. The drug…

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A Study Highlights the Benefit of a New Treatment for COPD. Could it Work in Rare Lung Diseases as Well?
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A Study Highlights the Benefit of a New Treatment for COPD. Could it Work in Rare Lung Diseases as Well?

According to a story from bioportfolio.com, a recent study has demonstrated the effectiveness of Trelegy Ellipta, a unique three component combination treatment, in reducing the effects of chronic obstructive pulmonary…

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Man With Soft Tissue Sarcoma is Refusing to Let His Diagnosis Spoil His Recent Marriage
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Man With Soft Tissue Sarcoma is Refusing to Let His Diagnosis Spoil His Recent Marriage

According to a story from the Peterborough Telegraph, James Willis received a disaster diagnosis on his first Valentine's Day after getting married at age 29. James had been worried about…

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