Mom Learns Baby Is One of 6 Born with Rare Disease — and All 5 Others Are Dead. Now, She's 'Terrified' for the Future (Exclusive)

Amanda LaValle pushed for her daughter to get genetic testing. When she learns that her daughter is one of only six people in ...
GlobalData on MSN

FDA approves UCB’s Kygevvi for genetic mitochondrial disease

The approval was based on data from a Phase II trial, two retrospective chart review studies and an expanded access programme ...

FDA Approves KYGEVVI® (doxecitine and doxribtimine), the First and Only Treatment for Adults and Children Living with Thymidine Kinase 2 deficiency (TK2d), a Rare an…

This novel therapy offers the first FDA-approved option to address the root cause of this ultra-rare condition, which causes ...
EurekAlert!

Eight babies born after Mitochondrial Donation treatment to reduce transmission of mitochondrial DNA disease

The UK’s pioneering licensed IVF technique to reduce the risk of mitochondrial diseases carried out in Newcastle has seen eight babies born, published research shows. All eight babies show no signs of ...
BioSpace

UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease

Kygevvi is indicated for patients with thymidine kinase 2 deficiency whose symptoms arise by 12 years of age. The disease ...

FDA approves first drug for Thymidine Kinase 2 Deficiency: All we know about this very rare mitochondrial disease

A groundbreaking FDA approval offers the first-ever treatment for ultra-rare Thymidine Kinase 2 Deficiency (TK2d). KYGEVVI, a ...
Isle of Wight County Press on MSN

Family's Christmas hamper aim for hospital's children's ward

The parents of an Isle of Wight child with a rare mitochondrial condition are asking for donations to provide Christmas ...
News Medical

Mitochondrial dysfunction in the blood-brain barrier may contribute to neuropsychiatric disease in 22qDS

In a new study led by the University of Pennsylvania School of Veterinary Medicine (Penn Vet) and Children's Hospital of Philadelphia (CHOP), researchers found that mitochondrial dysfunction in the ...
Labroots

Can a Drug Prevent Mitochondrial Diseases?

Mitochondria are incredible organelles. They not only provide crucial power to our cells, but these little machines also have their own tiny little genomes, and can even make their own proteins. But ...

FDA Approves KYGEVVI(R) (doxecitine and doxribtimine), the First and Only Treatment for Adults and Children Living with Thymidine Kinase 2 deficiency (TK2d), a Rare and ...

Hirano at Columbia University, an MDA Care Center, to develop a TK2-deficient mouse model, which proved that targeted treatments could restore mitochondrial function. This led to FDA granting ...