A “life changing” new therapy for an extremely rare medical condition which causes patients to grow a second skeleton has been proven successful in a key clinical trial. Relatives of people living with fibrodysplasia ossificans progressiva (FOP) say that they have “desperately waited” years for a medicine to “stop the disease ... A “life changing” new therapy for an extremely rare medical condition which causes patients to grow a second skeleton has been proven successful in a key clinical trial. Relatives of people living with fibrodysplasia ossificans progressiva (FOP) say that they have “desperately waited” years for a medicine to “stop the disease in its tracks”. Patients grow extra bones where they don’t belong, including within muscles and tendons A multi-country clinical trial has returned positive results for those with fibrodysplasia ossificans progressiva (Getty Images/iStockphoto) However, the new “disease-modifying” treatment has now been shown to cut new bone growth by over 90 per cent, decrease flare-ups and potentially provide renewed hope for surgical options in the future. FOP ranks among the world's rarest medical conditions, impacting just one in every million people. Because the disease is so rare, the clinical trial took place in 16 countries and regions, including the UK.

Original title: “Major breakthrough in treatment for ultra-rare 'second skeleton' disease fibrodysplasia ossificans progressiva | The Independent”

A “life changing” new therapy for an extremely rare medical condition which causes patients to grow a second skeleton has been proven successful in a key clinical trial. Relatives of people living with fibrodysplasia ossificans progressiva (FOP) say that they have “desperately waited” years for a medicine to “stop the disease ... A “life changing” new therapy for an extremely rare medical condition which causes patients to grow a second skeleton has been proven successful in a key clinical trial. Relatives of people living with fibrodysplasia ossificans progressiva (FOP) say that they have “desperately waited” years for a medicine to “stop the disease in its tracks”. Patients grow extra bones where they don’t belong, including within muscles and tendons A multi-country clinical trial has returned positive results for those with fibrodysplasia ossificans progressiva (Getty Images/iStockphoto) However, the new “disease-modifying” treatment has now been shown to cut new bone growth by over 90 per cent, decrease flare-ups and potentially provide renewed hope for surgical options in the future. FOP ranks among the world's rarest medical conditions, impacting just one in every million people. Because the disease is so rare, the clinical trial took place in 16 countries and regions, including the UK.

Original title: “Major breakthrough in treatment for ultra-rare 'second skeleton' disease”

DelveInsight s Pneumococcal Infections Pipeline Insight 2026 report provides comprehensive global coverage of available marketed and pipeline therapies for pneumococcal infections across various stages of clinical development The report provides insights into 18 companies and 20 pipeline drugs ... DelveInsight s Pneumococcal Infections Pipeline Insight 2026 report provides comprehensive global coverage of available marketed and pipeline therapies for pneumococcal infections across various stages of clinical development The report provides insights into 18 companies and 20 pipeline drugs covering clinical ... Press release - DelveInsight Business Research LLP - Pneumococcal Infections Clinical Trial Pipeline Analysis: 18+ Key Companies Shaping the Future of Pneumococcal Infections Therapeutics | DelveInsight - published on openPR.com The report evaluates emerging therapies by product type, clinical development stage, route of administration, and molecule type, while also highlighting inactive pipeline products and development activities. Key Takeaways from the Charcot-Marie-Tooth Disease Clinical Trial Landscape Report • The Treatment depends on the infection site, severity, patient characteristics, and local antimicrobial resistance patterns. Antibiotic therapy remains the cornerstone of treatment, while supportive care and vaccination are important components of pneumococcal disease management and prevention.

Original title: “Pneumococcal Infections Clinical Trial Pipeline Analysis: 18+ Key Companies Shaping the Future of Pneumococcal Infections Therapeutics | DelveInsight”

Kodiak Sciences will head to regulators with one of the candidates, an anti-VEGF antibody called zenkuda, after the late-stage results.

Original title: “Kodiak Sciences’ stock soars 166% after 2 assets match Eylea in Phase 3 trial”

The approval of Mirum Pharmaceuticals’ ALK2 inhibitor for fibrodysplasia ossificans progressiva on Friday adds a third option for patients who had long been without any treatments, joining Regeneron’s recently approved Pasatru and Ipsen’s Sohonos.

Original title: “FDA nod for Mirum’s Atebrioz makes 3 options for ultra-rare bone disease”

The FDA indicated that the data generated so far wouldn’t support an accelerated clearance of “I-DXd,” a setback that erases an edge Merck and Daiichi had over multiple fast-moving competitors.

Original title: “Merck, Daiichi end plans to seek speedy approval of lung cancer ADC”

Merck and Daiichi Sankyo’s withdrawal of an accelerated approval request for the antibody-drug conjugate ifinatamab deruxtecan is a “net positive” for rivals like GSK and Roche, according to RBC Capital Markets.

Original title: “Merck, Daiichi Sankyo retract accelerated approval request for lung cancer ADC”

Half of patients remained progression-free without maintenance

Original title: “Carvykti delivers five-year remissions in early myeloma”