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UCB wins CHMP favour for first thymidine kinase 2 deficiency treatment
04 Feb 2026 12:33 GMT
… patients with the ultra-rare mitochondrial disease in Europe.
The European Medicines … Europe”
The ultra-rare genetic mitochondrial disease causes progressive and severe muscle …
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Fundraising gala aims to raise awareness around mitochondrial disease
20 Jan 2026 18:32 GMT
… organization in 2024 after experiencing mitochondrial disease firsthand. Solomon’s organization is …
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Pharming Group announces 2026 financial guidance and highlights rare disease pipeline at Investor Day
03 Feb 2026 06:00 GMT
… dysregulation and mtDNA-driven mitochondrial disease
Introduces napazimone (KL1333) … mitochondrial DNA (mtDNA)‑driven mitochondrial disease. Fabrice Chouraqui, Chief Executive … (KL1333)– mtDNA-driven mitochondrial disease
Pharming management will outline the …
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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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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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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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