Threat to son's treatment for rare disease 'agony'
A mother has spoken about her "painful" experience over the potential end to her son's life-enhancing treatment for a rare ...
Live Science on MSN5h
POLG diseases: Rare genetic conditions that starve cells of energy and afflicted the late Prince of Luxembourg
POLG-related diseases disrupt the function of the mitochondria, or "powerhouses" of the cell — starving them of energy.
Fierce Pharma18m
Zevra Therapeutics embarks on diagnosis push as Miplyffa launch gains steam
It’s been just six months since Zevra Therapeutics scored an FD | Niemann-Pick disease type C therapy Miplyffa became ...
7-year-old in Hamilton has rare condition with $300K annual treatment her family can't afford
Cena Hermus, 7, was two years old when she was diagnosed with a rare, life-threatening disorder that causes severe obesity.
Polar Plunge in honor of Staten Islander with rare skin disease planned for this month
STATEN ISLAND, N.Y. — A fundraising Polar Plunge to honor John Hudson Dilgen will take place this month in South Beach to ...
Bruce Willis’s wife issues statement after deaths of Gene Hackman and Betsy Arakawa: ‘Caregivers need care, too’
His wife, as well as his daughters and ex-wife Demi Moore, have been praised for their candour discussing his condition, and ...
Primary school pupils brighten up Rare Disease Day with bold outfits
Pupils at Bridport Primary School put the spotlight on rare diseases by dressing in a riot of colour and learning about the challenges faced by those affected. As part of Rare Disease Day, children at ...
Director Who Worked With Prince Frederik Says it Was ‘Painful’ to Watch Him Struggle Before His Death
Filmmaker Mei Fa Tan reflected on her experience working with Luxembourg’s Prince Frederik before his death at the age of 22.
The Manila Times3h
Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results
Investors are encouraged to read the Company's Annual Report on Form 10-K when it is filed with the Securities and Exchange ...
Healio1h
Q&A: ‘An exciting breakthrough’: Gene transfer therapy promising in Danon disease
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
The American Journal of Managed Care2h
Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access
The study found that just 20% of trials submitted to both the FDA and European Medicines Agency had matching evidence.
Batten Disease: Parents fight for costly drug for children's rare disease to remain on NHS
Oliver and Amelia Carroll have Batten Disease - an incurable genetic disorder that causes rapid decline in a child's ability to walk, talk and see. | ITV News Granada ...