Why is "ADA-SCID" trending?

Search Interest Perspective

• Early identification of delayed-onset ADA deficiency: The case for expanded first-tier newborn screening - Rockefeller University Press
• Gene therapy delivers lasting immune protection in children with rare disorder - Newsroom | UCLA
• Experimental gene therapy treatment created at UCLA gives 'bubble girl' born with rare genetic disorder ADA-SCID a new life - ABC7 Los Angeles
• ADA-SCID Gene Therapy: Children Remain Cured After 10+ Years - Inside Precision Medicine
• Cell Therapy Weekly: Partnership to Advance Gene Therapy for ADA-SCID - RegMedNet
• Gene Therapy Shows Lasting Benefit for Children With Rare Disorder - MedPage Today
• Exposure to any germs could've killed her. 11 years later, she's living a normal life thanks to a gene therapy treatment. - NBC News
• Long-term outcomes of lentiviral gene therapy for ADA-deficient severe combined immunodeficiency - Contemporary Pediatrics
• The sustainability of gene therapies for rare diseases: a model born from ADA-SCID and WAS - Ricerca Malattie Genetiche Rare
• Gene therapy shows long-term success in children with rare immune disorder - News-Medical
• Gene therapy helps Virginia 12-year-old battle rare ‘bubble boy' disease - NBC4 Washington
• AGC Biologics Partners With Rarity PBC To Advance First U.S. Commercial Gene Therapy for ADA-SCID - BioPharma APAC
• A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID - Nature
• Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency - The New England Journal of Medicine
• Rarity PBC Raises $4.6 Million Seed Financing to Advance Gene Therapy for ADA-SCID - BioSpace
• An experimental therapy saves the lives of more than 60 children with a deadly disease - EL PAÍS English
• Donald Kohn awarded $14.7M CIRM grant to advance ADA-SCID gene therapy toward FDA approval - UCLA Health
• Fondazione Telethon and Orchard Therapeutics complete transfer of marketing authorization of Strimvelis for ADA-SCID in Europe - Ricerca Malattie Genetiche Rare
• Strimvelis receives European marketing authorisation to treat ADA-SCID - Ricerca Malattie Genetiche Rare
• Gene Therapy Provides Lasting Cure For 'Bubble Boy' Children - HealthDay
• Gene therapy is effective long-term in children with a serious rare disease - Science Media Centre España
• Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis- related graft failure and disseminated bacillus Calmette-Guérin infection - Haematologica
• Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients | Scientific Reports - Nature
• A Tripartite Alliance to Cure Seven Genetic Diseases - Ricerca Malattie Genetiche Rare
• Potential adverse reaction to gene therapy in a patient treated with Strimvelis for the treatment of ADA-SCID - Ricerca Malattie Genetiche Rare
• Orchard abandons promising gene therapy for rare immune disorder - BioPharma Dive
• Gene therapy restores immune function in children with rare immunodeficiency - National Institutes of Health (NIH) | (.gov)
• Orchard’s gene therapy improves overall survival in ADA-SCID trials - Clinical Trials Arena
• A case of severe combined immunodeficiency caused by adenosine deaminase deficiency with a new mutation - ScienceDirect.com
• Omenn Syndrome can Occur during Enzyme Therapy for Adenosine Deaminase Deficiency - Springer Nature Link
• Gene therapy for children born with rare immune disorder - National Institutes of Health (NIH) | (.gov)
• Gene therapy offers potential cure to children born without an immune system - Newsroom | UCLA
• ‘Bubble boy’ gene therapy reignites commercial interest - Nature
• ADA-SCID: 40 years of research set for valuation - Pharmaceutical Technology
• A decade after gene therapy, children born with deadly immune disorder remain healthy - Newsroom | UCLA
• Adenosine and Adenosine Deaminase Contrary Manifestations in Immunity - Wiley Online Library
• Kids with ‘bubble baby disease’ see gains after gene therapy - Nature
• Gene Therapy for ADA-SCID Still Benefitting Patients after Two to Three Years - Genetic Engineering and Biotechnology News
• Lentiviral gene therapy treatment for adenosine deaminase deficiency - 2 Minute Medicine
• Experimental gene therapy frees ‘bubble-boy’ babies from a life of isolation - Nature
• Severe Combined Immunodeficiency (SCID) and Its New Treatment Modalities - Cureus
• GSK receives positive CHMP opinion in Europe for Strimvelis™, the first gene therapy to treat very rare disease, ADA-SCID - GSK
• Revcovi (elapegademase-lvlr) for the treatment of ADA-SCID - Clinical Trials Arena
• As financial prospects for gene therapies dim, an ambitious charity picks up the torch - STAT
• Exploring Advances in ADA-SCID Gene Therapy: An Interview with Dr. Donald Kohn - Oncodaily
• Margaux discovering the world thanks to gene therapy - Ricerca Malattie Genetiche Rare
• Adenosine Deaminase (ADA) Deficiency: Report of Six New Cases and Reappraisal of Cutaneous Hypermelanosis as an Early Feature - Springer Nature Link
• Newborn screening strategy identifies otherwise missed adenosine deaminase deficiencies - Healio
• GSK, Fondazione Telethon and Ospedale San Raffaele announce EU regulatory submission for gene therapy to treat rare disease ADA-SCID - GSK
• Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy - Nature
• Human severe combined immunodeficiency: genetic, phenotypic, and functional diversity in one hundred eight infants - National Institutes of Health (NIH) | (.gov)
• Gene therapies for rare diseases are under threat. Scientists hope to save them - Nature
• FDA Approves Revcovi, a New Enzyme Replacement Therapy for the Treatment of ADA-SCID - drugdiscoverytrends.com
• A drug company abandoned a treatment for ‘bubble boy disease.’ After a 5-year fight, this little girl is about to get it - CNN
• Making History with the 1990 Gene Therapy Trial - Genetic Engineering and Biotechnology News
• Kids with 'bubble boy' disease could finally get access to a life-saving gene therapy, as a study starts back up at UCLA - Business Insider
• After life in a bubble, groundbreaking treatment opens Jakob Guziak’s world - UCLA Health
• Mutations in genes required for T-cell development: IL7R CD45 IL2RG JAK3 RAG1 RAG2 ARTEMIS , and ADA and severe combined immunodeficiency: HuGE review - Nature
• Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement - Nature
• Orchard Therapeutics' gene therapy Strimvelis linked to a leukemia case - Fierce Pharma
• GSK gives up on rare diseases as gene therapy gets two customers - Reuters
• Orchard raises $110M to bring gene therapy to market - Fierce Biotech
• Adenosine and Adenosine Deaminase Contrary Manifestations in Immunity - Wiley Online Library
• Gene therapy for severe combined immunodeficiencies and beyond | Journal of Experimental Medicine - Rockefeller University Press
• Gene therapy appears to cure “bubble boy disease” - Freethink
• Photo | Hussein before and after procedure - Newsroom | UCLA
• A Year After Approval, Gene-Therapy Cure Gets Its First Customer - MIT Technology Review
• NHS trust bids to take license for abandoned gene therapy - pharmaphorum
• Landmark clinical trial has ‘life changing’ results for Portlaoise boy - BreakingNews.ie
• U of A professor turns genetic marker for ‘bubble boy disease’ into music - University of Alberta
• Alabama screening newborns for two additional genetic disorders - WAFF
• Clonality analysis after retroviral-mediated gene transfer to CD34 - Nature
• NICE approves gene therapy for rare ‘bubble baby syndrome’ - The Pharma Letter
• Laois boy with no immune system has had ‘life-changing’ results in medical trial in London - Irish Independent
• Body, Cure Thyself - Discover Magazine
• Gene therapy breakthrough offers hope to children born without an immune system – The i Paper - The i Paper
• Elle's immune system is so severely compromised she has to live in a bubble with her 'bubble mummy' - ABC News & Headlines – Australian Broadcasting Corporation
• Hepatic Dysfunction as a Complication of Adenosine Deaminase Deficiency - The New England Journal of Medicine
• Children with no immune system get second chance in GOSH trial - London Evening Standard
• Newborn screening programme uncertain over availability of resources needed for expansion - Irish Medical Times
• Newborn screening: How a $10 test could have helped 'bubble' baby Isabelle - Nine.com.au
• Europe gives green light to first gene therapy for children - Guyana Chronicle
• Enzyme Replacement Therapy for Adenosine Deaminase Deficiency and Severe Combined Immunodeficiency - The New England Journal of Medicine
• Adenosine Deaminase-Severe Combined Immunodeficiency Market - openPR.com