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Discovery/Preclinical
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Gene therapy/gene editing combo could offer hope for some genetic disorders
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AmorChem Invests in Novel Approach to Target Myc-driven Cancers
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Researchers at Rush University Medical Center Find That Injecting Influenza Vaccines…
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Allergan’s Durysta Implant Approved to Treat Glaucoma
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Orphan-paediatric
Orphan-paediatric
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Orphan-paediatric
Synteract Launches Rare Disease Institute: An Innovational Program Specifically Focused on Rare Diseases
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February 27, 2020
Orphan-paediatric
Teva’s Austedo Fails a Pair of Tourette Syndrome Trials
Orphan-paediatric
Teva Announces Registration Trials of Deutetrabenazine in Pediatric Patients with Tourette Syndrome Did Not Meet the Primary Endpoint
Orphan-paediatric
Chiesi Group Announces Establishment of New Global Rare Diseases Division
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SUNFISH Pivotal Results for Risdiplam (RG7916) Demonstrate Medically Meaningful Benefit in Broadest Age Group of Patients Studied with Type 2 and 3 Spinal Muscular...
Orphan-paediatric
Columbus Children’s Foundation receives commitment to accelerate production of curative treatments...
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February 4, 2020
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Orphan-paediatric
Lonza Supporting Rocket Pharma with Clinical Manufacturing of LAD-I for the...
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January 28, 2020
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Orphan-paediatric
Rare disease of the immune system – Gene therapy success in...
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January 28, 2020
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Orphan-paediatric
Risdiplam (RG7916) Pivotal FIREFISH Study Demonstrated Statistically Significant Improvement in Infants...
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January 23, 2020
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Orphan-paediatric
Wave shares sink on disappointing results for WVE-120102 in Huntington’s disease...
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January 2, 2020
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