genetic disorders Archives

News and Trends 28 Jul 2023

FDA grants breakthrough therapy designation to Duchenne muscular dystrophy treatment

The U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy Designation to NS Pharma Inc.’s NS-089/NCNP-02, an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44…

July 28, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 18 Jul 2023

Boost for Charcot-Marie-Tooth patients as Novartis acquires DTx Pharma

DTx Pharma, a preclinical stage biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its fatty acid ligand conjugated oligonucleotide (FALCON) platform, has been acquired by Novartis. The FALCON…

July 18, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 23 Jun 2023

Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy

Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the treatment of ambulatory pediatric patients…

June 23, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 9 May 2023

Fabry disease treatment gets EU marketing authorization

Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc. have announced that the European Commission (EC) has granted marketing authorization to PRX-102 (pegunigalsidase alfa) in the European Union (EU) for the…

May 9, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 21 Apr 2023

Orphalan announces U.S. launch of drug to treat Wilson disease

Orphalan SA, an international orphan drug development and commercialization company, has announced the commercial launch in the U.S. for Cuvrior. Cuvrior is a new trientine tetrahydrochloride (TETA-4HCl). It is now…

April 21, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 28 Feb 2023

What’s happening on Rare Disease Day?

Rare Disease Day 2023 is on February 28. The day is dedicated to raising awareness for the approximately 300 million people around the world living with a rare disease. More…

February 28, 2023 - 8 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene…

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 3 Feb 2023

AI boosts genome editing

Researchers at the University of Zurich have developed a new tool that uses artificial intelligence to predict the efficacy of various genome-editing repair options. Unintentional errors in the correction of…

February 3, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 26 Jan 2023

Milestone for gene-edited therapy for sickle cell disease

Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics have announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application of exa-cel for the treatment of sickle cell disease (SCD)…

January 26, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 14 Dec 2022

NMD Pharma cleared for spinal muscular atrophy trial

NMD Pharma A/S has received clearance for its investigational new drug (IND) application from the US Food and Drug Administration (FDA) to advance NMD670 into a phase II clinical study…

December 14, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 8 Dec 2022

Metrion and KCNC1 Foundation announce collaboration to identify therapeutic

Metrion Biosciences Limited, and The KCNC1 Foundation are to collaborate to try and find a therapeutic for children. The KCNC1 Foundation is a not-for-profit organization focused on the development of…

December 8, 2022 - 2 minutesmins - By Liza Laws

News and Trends 17 Nov 2022

New mitochondria study could pave way for disease treatments

Mitochondria, the ‘powerhouses of the cell,’ are important cellular structures that are vital for their role of generating energy. They constantly fuse together and split apart. They contain a small…

November 17, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 16 Nov 2022

Enzymes could be key to understanding ‘origin of DNA errors’

Enzymes, critical to controlling how cells replicate in the human body, could be the very ingredient that encourages DNA to spontaneously mutate – causing potentially permanent genetic errors, according to…

November 16, 2022 - 3 minutesmins - By Jim Cornall

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