Brazil experiences paradox between advances in therapies for rare diseases and access barriers
Brazil is facing a paradox where advancements in precision medicine and gene therapies provide new treatment options for rare diseases, but access barriers remain.
‘Like a spelling mistake’: B.C. teen’s DNA ‘corrected’ to cure rare disease
A B.C. teenager, Ty Sperle, was successfully cured of chronic granulomatous disease through a groundbreaking medical procedure involving genetic correction.
Fiocruz starts studies with therapy for Spinal Muscular Atrophy; costs may reach 25% of current value
Fiocruz has initiated its first clinical study for a gene therapy aimed at treating severe Spinal Muscular Atrophy (SMA) in Brazil, potentially reducing treatment costs significantly.
Africa: Breakthrough Sickle Cell Gene Therapy a Distant Hope in Africa
A new gene therapy for sickle cell disease, successful in Western nations, remains unaffordable for most patients in Africa as Uganda begins nationwide newborn screening for the disorder.
‘Inbosa Incident’ Kolon Chairman Lee Woong-yeol Acquitted Again... "No Intent"
Lee Woong-yeol, chairman of Kolon, has been acquitted on appeal in a case related to the fraudulent misrepresentation of the components of the osteoarthritis gene therapy drug Inbosa.
Turning genes on and off. We are looking for the causes of cancer to treat it effectively
The article discusses the challenges of cancer treatment through gene therapy, highlighting that cancer is influenced by genetic mutations, age, and lifestyle rather than solely being a result of genetic mutations.
With the Wise One: When Gene Therapy Becomes a Hope for Life
The program highlights groundbreaking medical advancements in treating chronic and rare diseases, including FDA approval of a new daily oral medication for weight loss and a gene therapy for spinal muscular atrophy.
Sudanese family struggles to treat their son suffering from a rare genetic disease
The article highlights the struggles of a Sudanese family to obtain gene therapy for their son Imad, who is affected by Duchenne muscular dystrophy, a severe rare genetic disorder in children.
Gene therapy also for lowering cholesterol and changes in perceiving AI. Samuel Kováčik talks about what science can bring in 2026
In 2026, science will focus on AI development, with physicist Samuel Kováčik discussing possible significant changes and the uncertain direction they may take.