British-Finnish Alliance to Bring Gene Therapy to the Masses
Two European Bio-Tech companies are investigating the suitability of combining the two companies’ technology into one powerful gene therapy.
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GINA KOLATA, NY Times
- Oct 18, 2017
- 1 min
Gene Therapy Halts a Fatal Brain Disease
Patients with a genetic disease, adrenoleukodystrophy, or ALD, causing brain cell death were successfully treated with gene therapy.
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Diana Kwon, The Scientist
- Sep 1, 2017
- 1 min
CRISPR Restores Muscle Function in Mice
Using CRISPR, researchers have successfully treated congenital muscular dystrophy type 1A (MDC1A), a rare disease that can lead to severe mu
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Ben Locwin, Genetic Literacy Project
- Sep 1, 2017
- 1 min
Rare genetic diseases can arise from unsuspecting carriers
COMMAD is a syndrome which presents, ocular holes, dense bones, small eyes, large head, albinism, and deafness. A rare example of a disease
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PRWEB
- Sep 1, 2017
- 1 min
Introducing 123Genetix --- A New Non-Profit Organization Planning to Use Predictive Computer Models
123Genetix (www.123genetix.com) is developing a big data/deep learning solution to assist researchers: Develop new hypotheses; Design and ru
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Admin
- Sep 1, 2017
- 1 min
Preliminary Phase 2 Data from Spinal Muscular Atrophy Program Presented at CureSMA Conference
PTC Therapeutics (NASDAQ: PTCT) is a global biopharmaceutical company focused on the discovery, development, and commercialization of novel
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ROSE CAHALAN, Alcalde
- Sep 1, 2017
- 1 min
Parents of child with rare GM1 Lysosomal Storage Disease Lead Search for Treatment
When the Bragg family learned their daughter, Clara had GM1, a rare Lysosomal Storage Disease they, like many parents with children with rar
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Admin
- Aug 18, 2017
- 1 min
NIH scientists find rare disease clues in cell’s recycling system
Research carried out on a Niemann-Pick type C1 (NPC1) treatment can lead to a new generation of potential therapies for NPC1 and other simil
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Mathew Shanley, www.raredr.com
- Jun 25, 2017
- 1 min
New Data Provide Beneficial Evidence of Soliris (eculizumab) in PNH Patients
Alexion Pharmaceuticals, Inc. presented new data displaying benefits of Soliris (eculizumab) treatment for patients with PNH, regardless of
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Mathew Shanley, www.raredr.com
- Jun 25, 2017
- 1 min
Seattle Genetics announced promising results from its Phase 3 ECHELON-1 in Frontline Advanced Hodgki
Takeda and Seattle Genetics announced promising results from its Phase 3 ECHELON-1 Clinical Trial that evaluated Adcetris in Frontline Advan
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