Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders
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Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders

According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…

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First-of-its-Kind Study of Charcot-Marie-Tooth Disease Type 4J to Begin at UT Southwestern Med Center
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First-of-its-Kind Study of Charcot-Marie-Tooth Disease Type 4J to Begin at UT Southwestern Med Center

According to a publication from Charcot-Marie-Tooth News, the first patient has been enrolled in the first ever natural history study of type 4J Charcot-Marie-Tooth disease. Study collaborators — biotechnology company…

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Failure of Promising Investigatory Lupus Drug Prompts Discussion to Improve Clinical Trials

According to a publication from The Rheumatologist, the recent failure of a seemingly-promising lupus treatment in a phase 2 study has led to a discipline-wide discussion about new research and…

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First Patient Dosed in Phase 3 Study of Experimental Idiopathic Pulmonary Fibrosis Drug
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First Patient Dosed in Phase 3 Study of Experimental Idiopathic Pulmonary Fibrosis Drug

According to a press release from the San Francisco-based FibroGen, Inc., the first patient has been dosed in the Company's phase 3 clinical study of the experimental drug pamrevlumab for…

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Boston Children’s Hospital Studying Gene Therapy’s Potential for Treating CPVT Arrhythmia
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Boston Children’s Hospital Studying Gene Therapy’s Potential for Treating CPVT Arrhythmia

According to a publication from Fierce Biotech, researchers from Boston Children's Hospital have created a new gene therapy technique they believe could provide benefits to patients with inherited forms of…

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2019 United Leukodystrophy Conference Highlights

Patient Worthy had the privilege of attending another United Leukodystrophy Conference. The ULF’s 2019 event took place at the end of June in Illinois and had a record setting number…

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