Showing 1 to 12 of 32 results
Extensive Human Genome Sequencing Reveals Over 167,000 Structural Variants
Researchers sequenced 1,019 human genomes from diverse populations, discovering over 167,000 structural variants—double the previously known amount—improving understanding of rare diseases and cancer, and offering insights into human evolution and personalized medicine.
Extensive Human Genome Sequencing Reveals Over 167,000 Structural Variants
Researchers sequenced 1,019 human genomes from diverse populations, discovering over 167,000 structural variants—double the previously known amount—improving understanding of rare diseases and cancer, and offering insights into human evolution and personalized medicine.
Progress
44% Bias Score
FDA Rejects Promising Drug for Rare Mitochondrial Diseases
The FDA rejected elamipretide, a drug showing promise for treating rare mitochondrial diseases like MLS syndrome, despite positive patient testimonials and an advisory committee's narrow recommendation, raising concerns about the future of drug development for ultra-rare conditions.
FDA Rejects Promising Drug for Rare Mitochondrial Diseases
The FDA rejected elamipretide, a drug showing promise for treating rare mitochondrial diseases like MLS syndrome, despite positive patient testimonials and an advisory committee's narrow recommendation, raising concerns about the future of drug development for ultra-rare conditions.
Progress
48% Bias Score
Death of Russian Woman Highlights Healthcare System Failures
Tatiana Drozdova, a 23-year-old Russian woman with ADA-TKIN, died after a protracted legal battle to secure a 50 million ruble drug that was not delivered in time, highlighting systemic failures in healthcare access.
Death of Russian Woman Highlights Healthcare System Failures
Tatiana Drozdova, a 23-year-old Russian woman with ADA-TKIN, died after a protracted legal battle to secure a 50 million ruble drug that was not delivered in time, highlighting systemic failures in healthcare access.
Progress
56% Bias Score
Gene Editing Successfully Treats Baby's Life-Threatening Disorder
Doctors successfully treated a baby born with a life-threatening urea cycle disorder using a custom gene-editing therapy in Philadelphia, marking a major breakthrough in personalized medicine; the therapy appears to be working, allowing the baby to eat more protein, gain weight, and reach developmen...
Gene Editing Successfully Treats Baby's Life-Threatening Disorder
Doctors successfully treated a baby born with a life-threatening urea cycle disorder using a custom gene-editing therapy in Philadelphia, marking a major breakthrough in personalized medicine; the therapy appears to be working, allowing the baby to eat more protein, gain weight, and reach developmen...
Progress
40% Bias Score
Gene Editing Successfully Treats Rare Genetic Disease in Infant
A baby, K.J. Muldoon, born with severe CPS1 deficiency, a rare genetic disease affecting approximately one in a million infants, is thriving after receiving a custom CRISPR-based gene-editing therapy developed by the Children's Hospital of Philadelphia and Penn Medicine, offering hope for millions w...
Gene Editing Successfully Treats Rare Genetic Disease in Infant
A baby, K.J. Muldoon, born with severe CPS1 deficiency, a rare genetic disease affecting approximately one in a million infants, is thriving after receiving a custom CRISPR-based gene-editing therapy developed by the Children's Hospital of Philadelphia and Penn Medicine, offering hope for millions w...
Progress
48% Bias Score
Personalized Gene Therapy Shows Promise in Treating Baby with Rare Metabolic Disorder
In February 2025, a baby born with a life-threatening metabolic disorder received a personalized gene therapy in the US, showing significant health improvements and enabling the baby to return home, representing a potential breakthrough for treating rare genetic diseases with personalized base editi...
Personalized Gene Therapy Shows Promise in Treating Baby with Rare Metabolic Disorder
In February 2025, a baby born with a life-threatening metabolic disorder received a personalized gene therapy in the US, showing significant health improvements and enabling the baby to return home, representing a potential breakthrough for treating rare genetic diseases with personalized base editi...
Progress
40% Bias Score
UK and Florida Launch Nationwide Newborn Genomic Screening Programs
The UK's NHS will offer whole genome sequencing to all newborns by 2030, a £650 million initiative, while Florida's Sunshine Genetics Act launches a statewide genomic screening pilot program with a $3 million allocation, marking a major shift in public health towards proactive disease prevention and...
UK and Florida Launch Nationwide Newborn Genomic Screening Programs
The UK's NHS will offer whole genome sequencing to all newborns by 2030, a £650 million initiative, while Florida's Sunshine Genetics Act launches a statewide genomic screening pilot program with a $3 million allocation, marking a major shift in public health towards proactive disease prevention and...
Progress
40% Bias Score
Esteve Acquires Caprelsa, Advancing Specialized Therapy Strategy
Esteve, a Barcelona-based pharmaceutical company, announced the acquisition of Caprelsa, a thyroid cancer treatment, from Sanofi on Monday, furthering its strategy to specialize in highly specialized therapies, fueled by a €1 billion investment from Lubea in 2023. This follows several other acquisit...
Esteve Acquires Caprelsa, Advancing Specialized Therapy Strategy
Esteve, a Barcelona-based pharmaceutical company, announced the acquisition of Caprelsa, a thyroid cancer treatment, from Sanofi on Monday, furthering its strategy to specialize in highly specialized therapies, fueled by a €1 billion investment from Lubea in 2023. This follows several other acquisit...
Progress
32% Bias Score
Sanofi Acquires Blueprint Medicines for $9.1 Billion to Expand Rare Disease Portfolio
Sanofi is acquiring Blueprint Medicines Corporation for $9.1 billion, gaining access to Ayvakit/Ayvakyt, the only approved treatment for systemic mastocytosis, a rare disease, to strengthen its immunology portfolio and further its global leadership in this area.
Sanofi Acquires Blueprint Medicines for $9.1 Billion to Expand Rare Disease Portfolio
Sanofi is acquiring Blueprint Medicines Corporation for $9.1 billion, gaining access to Ayvakit/Ayvakyt, the only approved treatment for systemic mastocytosis, a rare disease, to strengthen its immunology portfolio and further its global leadership in this area.
Progress
44% Bias Score
Successful Gene Editing Treats Rare Genetic Disease in Baby
A baby with severe CPS1 deficiency, a rare genetic disorder affecting approximately one in a million infants, received a custom gene-editing therapy using CRISPR base editing, resulting in significant health improvements within months, opening possibilities for treating other rare genetic diseases.
Successful Gene Editing Treats Rare Genetic Disease in Baby
A baby with severe CPS1 deficiency, a rare genetic disorder affecting approximately one in a million infants, received a custom gene-editing therapy using CRISPR base editing, resulting in significant health improvements within months, opening possibilities for treating other rare genetic diseases.
Progress
40% Bias Score
Successful Gene Editing Treatment for Rare Genetic Disease
A 9.5-month-old baby, KJ Muldoon, successfully received a custom CRISPR-based gene editing therapy for severe CPS1 deficiency, a rare genetic disease affecting approximately one in a million babies. The treatment significantly improved his health, demonstrating the potential of personalized gene the...
Successful Gene Editing Treatment for Rare Genetic Disease
A 9.5-month-old baby, KJ Muldoon, successfully received a custom CRISPR-based gene editing therapy for severe CPS1 deficiency, a rare genetic disease affecting approximately one in a million babies. The treatment significantly improved his health, demonstrating the potential of personalized gene the...
Progress
40% Bias Score
Gene Editing Therapy Shows Promise in Treating Rare Genetic Disorder
Nine-month-old KJ Muldoon, diagnosed with severe CPS1 deficiency, a rare genetic disorder affecting one in a million babies, is thriving after receiving a custom CRISPR-based gene editing therapy developed in six months by researchers at Children's Hospital of Philadelphia and Penn Medicine.
Gene Editing Therapy Shows Promise in Treating Rare Genetic Disorder
Nine-month-old KJ Muldoon, diagnosed with severe CPS1 deficiency, a rare genetic disorder affecting one in a million babies, is thriving after receiving a custom CRISPR-based gene editing therapy developed in six months by researchers at Children's Hospital of Philadelphia and Penn Medicine.
Progress
40% Bias Score
Showing 1 to 12 of 32 results