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Science & Technology

A total of 135 posts are filed under Science & Technology
Epidermolysis Bullosa
New Hope For Epidermolysis Bullosa Patients with Investigational Therapy from Castle Creek Pharmaceuticals
Castle Creek Pharmaceuticals (CCP), a global company dedicated to delivering transformative therapies to patients with dermatologic and… Continue Reading
Sigma-Tau Pharmaceuticals, Inc. Changes Name to Leadiant Biosciences, Inc. And Reaffirms its Commitment to Rare Disease Communities
Sigma-Tau Pharmaceuticals, Inc., a leader in the development and commercialization of medicines for patients with rare diseases, today… Continue Reading
Aldeyra Therapeutics Launches the Aldeyra Registry for Patients with Sjögren-Larsson Syndrome
Aldeyra Therapeutics, Inc., a biotechnology company focused on the development of products to treat diseases related to aldehydes, today… Continue Reading
CureDuchenne Applauds the FDA Approval of Marathon’s EMFLAZA (deflazacort) for the Treatment of Duchenne Muscular Dystrophy
EMFLAZA is the First Drug in the U.S. Approved for Use by Patients Age Five and Older with Duchenne Regardless of Genetic Mutation… Continue Reading
Sanofi Genzyme’s Phase 2 Clinical Trial to Evaluate Oral Substrate Reduction Therapy for Gaucher Disease Type 3
Sanofi Genzyme is investigating an orally administered substrate reduction therapy in Gaucher disease type 3 as part of the 2-part LEAP… Continue Reading
PreventionGenetics Presents New Genetic Tests to Answer Questions on Stillbirths, Miscarriages, and Neonatal Death
Experiencing a miscarriage, stillbirth, or neonatal death can be devastating. It can leave parents with a lot of questions, like will it… Continue Reading
Penn Medicine Explores ResearchKit’s Potential for Studying Rare Disease
[SOURCE: Mobi Health News] In a first for the University of Pennsylvania Health System, Penn Medicine launched a new ResearchKit app today… Continue Reading