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- Rare Diseases

Rare Diseases Newsletter

'We have tears of joy' - Barnsley teen with ultra-rare disease set for US medical trial 27 Jan 2026 07:54 GMT
Daisy was diagnosed with an ultra-rare neurological disease at just 14(Image: GoFundMe) A South Yorkshire girl who has been diagnosed with an aggressive and ultra-rare neurological disease that will eventually leave her unable to walk is set to travel to …
Solon Springs musher, Canine Genetics Lab collaborate to research a rare disease 27 Jan 2026 02:59 GMT
… those sled dogs has a rare disease, and his owner has been …
Butler County community rallying to help 11-year-old fighting rare disease 27 Jan 2026 01:09 GMT
<![CDATA[Mirum Pharmaceuticals Completes $268.6 Million Acquisition of Bluejay Therapeutics to Strengthen Rare Disease Pipeline ]]> 26 Jan 2026 16:29 GMT
… within our core strengths in rare disease and builds on our deep … Therapeutics, Expanding Global Leadership in Rare Disease Mirum Pharmaceuticals January 26, 2026 … -Therapeutics-Expanding-Global-Leadership-in-Rare-Disease
Iran’s breakthrough oral SMA drug sparks hope for rare disease patients worldwide 26 Jan 2026 15:20 GMT
… watched as modern treatments for rare diseases like SMA remained out of … , including children and patients with rare diseases, from external pressures. The successful …
Mirum Pharmaceuticals Completes Acquisition of Bluejay Therapeutics, Expanding Global Leadership in Rare Disease 26 Jan 2026 14:58 GMT
… Inc. (NASDAQ: MIRM), a leading rare disease company, today announced the successful … overlooked conditions, Mirum combines deep rare disease expertise with strong connections to … and purposeful collaboration across the rare disease ecosystem. Learn more at …
Tot saved with just 24 hours to spare after 'tonsillitis' turned out to be rare disease 26 Jan 2026 14:54 GMT
… actually come down with a rare disease that in some cases, can …
Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, and Advocacy 26 Jan 2026 14:00 GMT
… ensuring families are never navigating rare disease alone. Strength in Community Stories … address challenges of living with rare diseases and provide resources and … closer to future treatments. For rare diseases, progress depends on sustained scientific …
Scots toddler's rare disease spotted with 24 hours to spare 26 Jan 2026 12:23 GMT
… of a toddler with a rare disease has called for greater awareness …
My toddler's rare disease was spotted with 24 hours to spare 26 Jan 2026 06:33 GMT
… a toddler diagnosed with a rare disease has called for greater awareness …

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