Interview with Dr. Michelle Krishnan: New Therapies for Neurodevelopmental Disorders
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Interview with Dr. Michelle Krishnan: New Therapies for Neurodevelopmental Disorders

Front Line Genomics has recently interviewed Dr. Michelle Krishnan, who is the Translational Medicine Leader in Rare Diseases at Roche. She focuses on rare neurodevelopmental disorders, in which she works…

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With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?
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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?

According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…

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ICYMI: The Manufacturer of Spinal Muscular Atrophy Drug Zolgensma has Made a Lottery for Life
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ICYMI: The Manufacturer of Spinal Muscular Atrophy Drug Zolgensma has Made a Lottery for Life

As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when…

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Drugmaker Novartis Launches Lottery for Access to Critical Spinal Muscular Atrophy Treatment
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Drugmaker Novartis Launches Lottery for Access to Critical Spinal Muscular Atrophy Treatment

According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each…

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Parents React to Novartis’ International “Baby Lottery” for Zolgensma with Hope But Also With Outrage

According to a recent article in Euronews, this year one hundred children who were diagnosed with spinal muscular atrophy (SMA) will receive Zolgensma, the world’s highest-priced drug, at no cost.…

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A New Pediatric Precision Medicine Center is Opening in Utah
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A New Pediatric Precision Medicine Center is Opening in Utah

A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…

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Queensland Family Calls for Spinal Muscular Atrophy Newborn Screening After Their Daughter was Diagnosed
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Queensland Family Calls for Spinal Muscular Atrophy Newborn Screening After Their Daughter was Diagnosed

According to a story from abc.net.au, parents Kellee and Jamie Clarkson of Queensland, Australia are calling for the implementation of newborn screening in the state for the rare disorder spinal…

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Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients
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Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients

According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…

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Personalized Medicine: A Drug Tailored for a Six Year Old With Batten Disease May Serve as a Template for Similar Rare Disorders

  Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…

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Clinical Trials Show Promising Results for Risdiplam in Treatment of Spinal Muscular Atrophy Types 1, 2, and 3

According to a recent article in SMA News Today, risdiplam (formerly RG7916)  is in ongoing studies of patients with spinal muscular atrophy (SMA). The drug is an investigational medicine developed by Genentech…

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This Doctor’s Life Changed Forever When His Son Was Born With Spinal Muscular Atrophy
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This Doctor’s Life Changed Forever When His Son Was Born With Spinal Muscular Atrophy

According to a story from medicalxpress.com, Dr. Nathan Hoot is an emergency medical physician, but his role as a doctor doesn't entirely end when he leaves the hospital because his…

Continue Reading This Doctor’s Life Changed Forever When His Son Was Born With Spinal Muscular Atrophy