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May 18, 2018May 18, 2018

Mitochondrial Disease Treatment Candidate KH176 Shows Potential, Study Finds

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Khondrion’s mitochondrial disease treatment candidate KH176 has potential to be effective in a broad range of disorders, a new study reports. The research, “KH176 Safeguards Mitochondrial Diseased Cells from ... Read more

May 17, 2018May 17, 2018

‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

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The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. ... Read more

May 17, 2018May 17, 2018

#ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

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The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

May 11, 2018May 11, 2018

Multiple Consultations, Tests and Misdiagnoses Mark Mitochondrial Patients ‘Odyssey,’ Survey Finds

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People with mitochondrial disease typically can visit on average eight different doctors, and undergo multiple tests before finally getting an accurate diagnosis, a patients’ survey reports. The study reporting ... Read more

May 4, 2018May 4, 2018

FDA Grants NeuroVive’s KL1333 Orphan Drug Status for Treatment of Mitochondrial Diseases

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Investigational compound KL1333 was granted orphan drug status by the U.S. Food and Drug Administration for the treatment of inherited mitochondrial respiratory chain diseases, NeuroVive Pharmaceutical AB recently announced. The designation is a ... Read more

May 2, 2018May 2, 2018

#ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

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At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan ... Read more

April 20, 2018April 20, 2018

#AAN2018 – Mutation in USMG5 Gene Causes Leigh Syndrome, Study Shows

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Researchers found that a mutation in the USMG5 gene causes Leigh syndrome and deficient energy production in cells. This is the first evidence linking alterations in this gene to ... Read more

April 13, 2018April 13, 2018

Some Antioxidants Show Benefits in Models of Mitochondrial Disease, Study Reports

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The antioxidants vitamin E and N-acetylcysteine (NAC) prolonged survival and protected against brain damage in models of mitochondrial disease, a study shows. The compounds will now be tested in clinical ... Read more

April 13, 2018April 12, 2018

NIH Agency Pioneers Collaborative Research into Rare Diseases

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A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. ... Read more

April 6, 2018April 6, 2018

Genetics Foundation to Donate Adapted Bikes to Charlotte-Area Children with Genetic Conditions During Day of Caring

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Children living with genetic or mitochondrial disease in Charlotte, North Carolina, will soon be able to ride new adapted bikes donated by the ACMG Foundation for Genetic and Genomic ... Read more

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