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Oct 18, 2017
1 min

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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Gene Therapy Halts a Fatal Brain Disease

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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Rare genetic diseases can arise from unsuspecting carriers

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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Introducing 123Genetix --- A New Non-Profit Organization Planning to Use Predictive Computer Models

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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Preliminary Phase 2 Data from Spinal Muscular Atrophy Program Presented at CureSMA Conference

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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Parents of child with rare GM1 Lysosomal Storage Disease Lead Search for Treatment

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

5 views0 comments

NIH scientists find rare disease clues in cell’s recycling system

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

20 views0 comments

New Data Provide Beneficial Evidence of Soliris (eculizumab) in PNH Patients

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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Seattle Genetics announced promising results from its Phase 3 ECHELON-1 in Frontline Advanced Hodgki

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

19 views0 comments

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