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GenSight Soars After Securing Compassionate Use Authorization in France
22 Dec 2025 12:59 GMT
… neuropathy (LHON) is a rare mitochondrial disease, maternally inherited, characterized by the …
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UCB Drug Becomes First FDA-Approved Treatment for Ultra-Rare and Fatal Mitochondrial Disease
05 Nov 2025 22:43 GMT
The energy that powers cells of the human body comes from tiny cellular components called mitochondria. Just as a draining battery makes it difficult to operate an electronic device, mitochondrial disorders impair cellular function and cause progressively …
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UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease
04 Nov 2025 13:36 GMT
… and CEO of the United Mitochondrial Disease Foundation, said in a statement …
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FDA approves UCB’s Kygevvi for genetic mitochondrial disease
04 Nov 2025 11:11 GMT
… and doxribtimine) to treat genetic mitochondrial disease, thymidine kinase 2 deficiency (TK2d … options for this rare genetic mitochondrial disease beyond supportive [palliative] care.
“We …
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FDA approves first drug for Thymidine Kinase 2 Deficiency: All we know about this very rare mitochondrial disease
04 Nov 2025 06:04 GMT
… second approved treatment for a mitochondrial disease in the US this year … ).Besides, for the field of mitochondrial disease, this sets an important precedent …
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FDA Approves KYGEVVI® (doxecitine and doxribtimine), the First and Only Treatment for Adults and Children Living with Thymidine Kinase 2 deficiency (TK2d), a Rare and Devastating Mitochondrial Disease
03 Nov 2025 22:25 GMT
… MDA. “Our longstanding investment in mitochondrial
disease research and clinical trial support …
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Tisento Therapeutics Announces Publication of MELAS Interview Study, Incorporating Patients’ Perspectives to Elucidate Symptoms and Impacts of Rare Mitochondrial Disease
03 Nov 2025 12:00 GMT
… -life impacts of this rare mitochondrial disease. The publication, entitled “Signs, Symptoms … enrolling approximately 44 participants at mitochondrial disease centers of excellence in the …
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Muskingum County Commissioners Recognize Mitochondrial Disease Awareness Week
15 Sep 2025 23:34 GMT
ZANESVILLE, OH – September is Mitochondrial Disease Awareness Month.
To highlight and … to go to the United Mitochondrial Disease Foundation (UMDF) through the Bet …
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Tom Andrews: 21yo with rare mitochondrial disease debuts abstract paintings in solo Subiaco exhibition
17 Sep 2025 18:55 GMT
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The Lily Foundation celebrates approval of first ever mitochondrial disease treatment by NICE
14 Aug 2025 11:05 GMT
… Foundation, the UK’s leading mitochondrial disease charity, is today celebrating a … throughout the NICE evaluation process.
Mitochondrial disease is caused by faults in … the most common types of mitochondrial disease, primarily affecting the optic nerve …
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