#rare-diseases

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#gene-therapy
Parenting
fromwww.standard.co.uk
2 months ago

Gene therapy experiment gives children 'life-changing' sight boost at London hospital

Pioneering gene therapy restores sight in young children with a rare genetic condition.
Parenting
fromwww.standard.co.uk
2 months ago

Gene therapy experiment gives children 'life-changing' sight boost at London hospital

Pioneering gene therapy restores sight in young children with a rare genetic condition.
SF parents
fromenglish.elpais.com
2 weeks ago

A Spanish therapy saves the lives of the unluckiest family in the world'

A revolutionary gene therapy has transformed the lives of children suffering from a rare genetic disorder caused by a DNA mutation.
Miscellaneous
fromenglish.elpais.com
3 months ago

The second revolution in gene therapy that has cured Javier, a child with a lethal error in the X chromosome

A small sign led to diagnosis of Wiskott-Aldrich syndrome, which was overcome through groundbreaking gene therapy.
more#gene-therapy
US news
fromBoston.com
1 week ago

Baby is healed with world's first personalized gene-editing treatment

A custom gene-editing treatment for a rare genetic disorder may pave the way for therapies for thousands of other genetic diseases.
fromSilicon Canals
1 week ago

Dutch-based Azafaros raises 132M

Azafaros has been impressive in its execution with nizubaglustat poised to begin Phase 3 clinical development and the potential to significantly improve the lives of NPC and GM1/GM2 patients.
Startup companies
fromwww.independent.co.uk
3 weeks ago

Rare diseases often go undiagnosed or untreated in parts of Africa. A project seeks to change that

At home, Lam and her husband Pathe smiled over an old video clip of their daughter beaming as she celebrated her 13th birthday with cake and sparklers.
OMG science
OMG science
fromNature
1 month ago

Whole-genome sequencing susses out rare diseases

Whole-genome sequencing offers a more comprehensive solution for diagnosing rare genetic diseases than traditional exome sequencing.
#patient-advocacy
OMG science
fromwww.nytimes.com
2 months ago

Drugs Have Uses We Can't Imagine. He's Using A.I. to Find Them.

AI-driven treatment options can significantly impact the care of patients with rare diseases.
Persistence in seeking unconventional solutions can lead to unexpected and life-saving outcomes.
OMG science
fromwww.nytimes.com
2 months ago

Drugs Have Uses We Can't Imagine. He's Using A.I. to Find Them.

AI-driven treatment options can significantly impact the care of patients with rare diseases.
Persistence in seeking unconventional solutions can lead to unexpected and life-saving outcomes.
more#patient-advocacy
Education
fromIrish Independent
2 months ago

Dublin girl with rare skin disease excited to go to secondary school

Casey Connors, 12, is preparing for secondary school while managing her condition, epidermolysis bullosa, with strong family and community support.
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