Company Receives Clearance to Conduct Trial for Propionic and Methylmalonic Acidemia
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Company Receives Clearance to Conduct Trial for Propionic and Methylmalonic Acidemia

According to a press release from HemoShear Therapeutics, the company has recently received official clearance from the US Food and Drug Administration (FDA) in regards to its Investigational New Drug…

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New Treatment Approved in Canada for Gastrointestinal Stromal Tumor
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New Treatment Approved in Canada for Gastrointestinal Stromal Tumor

According to a story from Biotech 365, the biopharmaceutical company Deciphera Pharmaceuticals has recently announced that the company's drug ripretinib (marketed as QINLOCK™) has been approved by Health Canada. The…

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The First Treatment for Adult-Onset Still’s Disease Just Got FDA Approved
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The First Treatment for Adult-Onset Still’s Disease Just Got FDA Approved

According to a story from PR Newswire, the US Food and Drug Administration (FDA) has just approved a treatment for rare adult-onset Still's disease for the first time ever. The…

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CRISPR Editing Appears Effective in Three Patients with Rare Diseases
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CRISPR Editing Appears Effective in Three Patients with Rare Diseases

According to a story from interestingengineering.com, the capabilities of CRIPSR gene editing technology have been creating a buzz in the medical field for years now, with many seeing the technology…

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June is Scleroderma Awareness Month: Spreading Rare Disease Awareness
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June is Scleroderma Awareness Month: Spreading Rare Disease Awareness

According to a story from Scleroderma News, June is recognized as Scleroderma Awareness Month. While physical distancing as a result of the ongoing coronavirus/COVID-19 pandemic may keep some folks at…

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On July 1, the Marfan Foundation and Loeys-Dietz Syndrome Foundation Will Officially Partner Up
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On July 1, the Marfan Foundation and Loeys-Dietz Syndrome Foundation Will Officially Partner Up

Education, research, support, and patient advocacy: these are just four of the amazing things that rare disease or disorder foundations offer. Many foundations seek to offer programs and resources to…

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FDA Approves a New Treatment for Non-Radiographic Axial Spondyloarthritis
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FDA Approves a New Treatment for Non-Radiographic Axial Spondyloarthritis

In a press release from Eli Lilly and Company, the company has announced recently that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (BLA)…

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Consumers with Allergies, EoE Worry When FDA Relaxes Food Labeling Guidelines
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Consumers with Allergies, EoE Worry When FDA Relaxes Food Labeling Guidelines

  Stacey Saiontz is many things: a worker, a wife, a mother. But right now, she is concerned. As an allergy parent, or a parent whose child has severe food…

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The Very First Treatment for Neurofibromatosis Type 1 Has Been Approved by the FDA

Neurofibromatosis  Neurofibromatosis is a rare condition that presents in different forms. Neurofibromatosis type one (NF1) causes tumors to grow on the plexiform neurofibromas, a type of nerve in the body. These tumors…

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Fabry Disease: A Biologics License Application for Pegunigalsidase Alfa has Been Submitted to the FDA
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Fabry Disease: A Biologics License Application for Pegunigalsidase Alfa has Been Submitted to the FDA

According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…

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May 16th is Hereditary Angioedema Day : Spreading Rare Disease Awareness
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May 16th is Hereditary Angioedema Day : Spreading Rare Disease Awareness

May 16th is celebrated annually as Hereditary Angioedema (HAE) Awareness Day. The event was established with the goal of spreading awareness about this rare condition among both the general public…

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