FDA Grants Rare Pediatric Disease Designation for WP1066
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FDA Grants Rare Pediatric Disease Designation for WP1066

The FDA has recently granted its Rare Pediatric Disease designation to Moleculin's WP1066 for three indications: atypical teratoid rhabdoid tumor, diffuse intrinsic pontine glioma, and medulloblastoma. This designation will aid…

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RDLA Webinar Provides Vital Updates on Legislation Relevant to the Rare Disease Community
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RDLA Webinar Provides Vital Updates on Legislation Relevant to the Rare Disease Community

On October 15th, 2020, the Rare Disease Legislative Advocates (RDLA) held a webinar discussing some of the latest news regarding ongoing legislation that is most relevant to the rare disease…

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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation
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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation

According to a story from BioSpace, Ziopharma Oncology, Inc. has recently announced that its investigational therapy Ad-RTS-hIL-12 with veledimex (also called Controlled IL-12) has been given Rare Pediatric Disease designation…

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A Teenager’s Plan to Help her Friend with Diffuse Intrinsic Pontine Glioma Takes Off
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A Teenager’s Plan to Help her Friend with Diffuse Intrinsic Pontine Glioma Takes Off

According to a story from Essex Live, Lily Wythe is a fourteen year old girl who was recently diagnosed with a rare form of brain cancer called diffuse intrinsic pontine…

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Recent Study Confirms Validity of the Therapeutic Target of an Experimental Brain Cancer Drug
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Recent Study Confirms Validity of the Therapeutic Target of an Experimental Brain Cancer Drug

According to a story from BioSpace, Oncotelic, Inc., recently announced the publication of a study in the Journal of Clinical Research in Pediatrics. This study appears to back up the therapeutic…

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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation
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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation

According to a story from Biotech 365, the immuno-oncology company Mateon Therapeutics Inc. has recently announced that the company's investigational product candidate OT101 has earned the US Food and Drug…

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New Drug Has Promise for Treating Diffuse Intrinsic Pontine Glioma and Fibrodysplasia Ossificans Progressiva

Diffuse Intrinsic Pontine Glioma Diffuse intrinsic pontine glioma (DIPG) is a rare form of childhood brain cancer that has a 100% mortality rate. Children diagnosed with DIPG only typically live…

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An Experimental Biopsy Approach Could Improve Diffuse Intrinsic Pontine Glioma Monitoring

According to a story from tgen.org, a team of scientists from the Translational Genomics Research Institute (TGEN) have recently initiated a study that will test the effectiveness of liquid biopsy…

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