European Study Identifies Sources of Delayed Diagnosis & More — This Week in RARE Daily

May 16, 2024

This Week in RARE Daily is a feature from Global Genes where you can get a quick rundown of the top 5 headlines in the rare disease space from our editorial staff. Here are top stories from this past week, May 10 – 16, 2024:

European Study Identifies Sources of Delayed Diagnosis
People with a rare disease in Europe saw an average of eight physicians and 4.7 years to get a diagnosis, according to a new study by rare disease patient organization Eurodis-Rare Diseases Europe.
Read Article

Ionis and Biogen Stop Development of ALS Drug After Trial Failure
Ionis Pharmaceuticals and Biogen said they were stopping development of BIIB105, an experimental antisense oligonucleotide for the treatment of the rare, neurodevelopmental condition amyotrophic lateral sclerosis, based on data from the phase 1/2 ALSpire study.
Read Article

Novo Nordisk’s Hemophilia A Candidate Hits Primary Endpoint in Phase 3 Study
Novo Nordisk reported strong positive results from the pivotal study of its experimental, next-generation prophylaxis for the rare bleeding disorder hemophilia A.
Read Article

Ambry and PacBio Selected for GREGoR Consortium Effort to Identify Rare Diseases
The University of California, Irvine and the GREGoR Consortium have selected clinical genomic testing company Ambry Genetics and genome sequencing company PacBio to support the Pediatric Mendelian Genomics Research Center program in its efforts to better understand the underlying biology of rare diseases.
Read Article

Centogene Secures $20 Million, Expanding Relationship with Saudi Biotech Lifera
Centogene entered into a set of agreements with Saudi Arabian biotech Lifera that expands their relationship and provides approximately $20 million to support its mission is to provide data-driven, life-changing answers to patients, physicians, and pharma companies for rare and neurodegenerative diseases.
Read Article

More from RARE Daily

Stay Connected

Sign up for updates straight to your inbox.