11 Year Old Aims to Spread Awareness about Primary Pulmonary Hypertension, a Rare Disease That has Ravaged Her Family
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11 Year Old Aims to Spread Awareness about Primary Pulmonary Hypertension, a Rare Disease That has Ravaged Her Family

According to a story from the Borehamwood & Elstree Times, the family of 11 year old girl Sivan Hermon is plagued by a rare disease: primary pulmonary hypertension. As the…

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A Teenager was Killed by a Disease so Rare it Didn’t Have a Name, But His Story Could Change the Lives of Others Like Him

According to a story from NBC News, Mitchell Herndon, a 19 year old from Missouri, recently passed away due to the progression of a rare genetic disease that is so…

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A Man with CIDP Slid 400 Feet Down the New River Gorge’s High-Line in a Wheelchair.
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A Man with CIDP Slid 400 Feet Down the New River Gorge’s High-Line in a Wheelchair.

As originally reported in WWVA, West Virginian Sib Weatherford spent one afternoon this fall doing what nobody though possible: he slid 400 feet down the New River Gorge's high-line in…

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Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia
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Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…

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A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her
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A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her

According to a story from Technology Review, the story of Mila Makovec shows the potential impact that personalized medicine could have on patients while also revealing some of its limitations.…

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Phase 3 Clinical Trial for Amyotrophic Lateral Sclerosis Has Completed Enrollment
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Phase 3 Clinical Trial for Amyotrophic Lateral Sclerosis Has Completed Enrollment

BrainStorm Cell Therapeutics has just announced that they have completed enrollment in their Phase 3 clinical trial for amyotrophic lateral sclerosis (ALS). Enrollment was initiated way back in October of…

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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes
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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes

According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…

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The FDA Has Just Approved the Very First Therapy for Erythropoietic Protoporphyria

EPP Erythropoietic protoporphyria (EPP) is a rare disease that causes extreme light sensitivity. This sensitivity is so extreme that being outside is extremely difficult for patients and many only come…

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Study Analyzes New Outcome Measure for Duchenne Muscular Dystrophy
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Study Analyzes New Outcome Measure for Duchenne Muscular Dystrophy

According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement…

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Company Awarded Grant to Conduct Amyotrophic Lateral Sclerosis Clinical Trial
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Company Awarded Grant to Conduct Amyotrophic Lateral Sclerosis Clinical Trial

According to a story from PR Newswire, the biopharmaceutical company Clene Nanomedicine, Inc., has issued an announcement in regards to its Australian subsidiary. This subsidiary has been awarded a grant…

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