Editor’s Choice: A Treatment for Mitochondrial Disease, Rare Pediatric Disease Designation in Osteosarcoma, and a “Chaotic” Birth

Happy Monday! This week, we have stories on a potential treatment approach for an aggressive form of mitochondrial disease, an experimental treatment for osteosarcoma earning Rare Pediatric Disease designation, and…

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Study of the Week: Scientists Discover a Potential Therapy for an Aggressive Form of Mitochondrial Disease
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Study of the Week: Scientists Discover a Potential Therapy for an Aggressive Form of Mitochondrial Disease

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

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Researchers Unearth Potential Therapy for FBXL4 Mitochondrial Disease
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Researchers Unearth Potential Therapy for FBXL4 Mitochondrial Disease

FBXL4-related mitochondrial disease urgently requires new therapies. Unfortunately, this condition is often fatal by early ages (early to mid-childhood). Therefore, it is important to spur research into potential interventions or…

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Multigenerational Mitochondrial Disease: Spreading Awareness
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Multigenerational Mitochondrial Disease: Spreading Awareness

  Written by Gina Baker This year Global Mitochondrial Disease Awareness week is September 19-25, but my family and I will continue to fight this battle for the rest of…

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Girl with Mitochondrial Disease Celebrates 18th Birthday Thanks to Doctors and Her Own Determination
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Girl with Mitochondrial Disease Celebrates 18th Birthday Thanks to Doctors and Her Own Determination

Mitochondrial Disease Mitochondrial disease is a rare and progressive condition that currently does not have a cure. The mitochondria is responsible for providing energy to the body. This energy is used…

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Study Reinforces the Importance of Vaccines for Congenital Lactic Acidosis Patients and Other Rare Diseases

A recent study published in PLOS ONE has reinforced the importance of vaccinations specifically for those already fighting a rare disease. This study was written by Sylvie Lesage from the…

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September 13-19 is Mitochondrial Disease Awareness Week: Spreading Rare Disease Awareness
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September 13-19 is Mitochondrial Disease Awareness Week: Spreading Rare Disease Awareness

The third week of September is recognized annually as Mitochondrial Disease Awareness Week. This global event is held in order to increase awareness of mitochondrial disease in the medical community…

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Mitochondrial Disease Patient’s Parents Lose Law Suit Against Four Doctors and Boston Children’s Hospital
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Mitochondrial Disease Patient’s Parents Lose Law Suit Against Four Doctors and Boston Children’s Hospital

  The Boston Globe recently interviewed the principals in a case brought by the Pelletier family against the Boston Children’s Hospital. The article gives details about a family’s charge of…

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Phase 2b Trial for Mitochondrial Diseases has Begun
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Phase 2b Trial for Mitochondrial Diseases has Begun

Khondrion has just announced that the very first patient has been dosed in their Phase 2b study for mitochondrial diseases called KHENERGYZE. This patient population has a high unmet need…

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“We Don’t Know Anything:” a Mitochondrial Encephalomyopathy Story From the Czech Republic
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“We Don’t Know Anything:” a Mitochondrial Encephalomyopathy Story From the Czech Republic

When Lucinka was born, it seemed like everything was all right. But it wasn’t. Lucinka wasn’t doing well, and a whirlwind of examinations began. First, a diagnosis couldn’t be made.…

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After a Childhood Plagued by Illness, This Woman Was Finally Diagnosed With Mitochondrial Disease
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After a Childhood Plagued by Illness, This Woman Was Finally Diagnosed With Mitochondrial Disease

According to a story from Wales Online, Samantha Josey's childhood was characterized by unpleasant symptoms and health problems, resulting in hospital visits and bouts of serious illness. Her symptoms included…

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Study to Test Possible Biomarker for Myopathy Associated With Mitochondrial Disease
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Study to Test Possible Biomarker for Myopathy Associated With Mitochondrial Disease

According to a story from clinicaltrials.gov, the Children's Hospital of Philadelphia is sponsoring a study that has the potential to make mitochondrial disease research and drug development much easier. The…

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Minovia Kicks Off Phase I/II Trial of Experimental Pearson Syndrome Treatment
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Minovia Kicks Off Phase I/II Trial of Experimental Pearson Syndrome Treatment

According to a publication from Business Wire, the Israeli biotechnology company Minovia Therapeutics recently finished dosing the first participant in a phase I/II clinical study of the Company's experimental mitochondrial…

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Study Identifies Broader Range of Clinical Presentation for Mitochondrial Disease
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Study Identifies Broader Range of Clinical Presentation for Mitochondrial Disease

According to a story from Wellcome Center Mitochondrial Research, a multi-center collaborative study that involved researchers from different sites around the UK has resulted in an improved understanding of the…

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