Rare Disease Report

Top Rare Disease Report Articles for September 2017

SEPTEMBER 29, 2017
1. New Data Showcases Long Term Use of Eculizumab in Myasthenia Gravis
The study found that patients who took eculizimab for 26 weeks in the REGAIN study and then continued to take the drug in the extension study (n=56), had stable scores on their myasthenia gravis activities of daily living (MG-ADL) questionnaire. 

Read the full article at: www.raredr.com/link/2661

2. George Yohrling, Ph.D Discusses Current and Potential HD Treatment Options
Since 2012, George Yohrling, PhD has served as the Senior Director, Mission and Scientific Affairs for the Huntington’s Disease Society of America (HDSA). In this video, he discusses current treatment options for patients, as well as therapies in the pipeline and why it’s such an exciting time to be a part of the Huntington’s Disease (HD) research community.

Read the full article at:  www.raredr.com/link/2649

3. Sancilio Gets Rare Pediatric Disease Designation for Sickle Cell Disease Treatment
Sancilio Pharmaceuticals Company, Inc. (SPCI) announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Altemia Soft Gelatin Capsules for the treatment of sickle cell disease (SCD) in children.

Read the full article at: www.raredr.com/link/2686

4. Avrobio Developing a Gene Therapy for Pompe Disease
This will be the third gene therapy the company has in its pipeline to treat a rare lysosomal storage disease; the other 2 programs are for Fabry disease and Gaucher disease.

Read the full article at:  www.raredr.com/link/2748

5. Pfizer Creates New Smaller Company to Focus on Rare Diseases
Big Pharma company Pfizer Inc. is taking a page out of some of the smaller biotech companies that it would normally acquire. Pfizer is creating a smaller company to develop orphan drugs.

Read the full article at: www.raredr.com/link/2747

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