At AMCP Nexus 2026, Susan M. Wescott, RPh, MBA, discussed how health plans evaluate rare disease therapies amid limited data and high costs. As more rare disease therapies come to market, health plans are often evaluating products for conditions they have never…
FDA draft guidance offers rare disease gene therapy developers clarity on personalized approvals, BLAs, and faster variant updates. Read more here. - The US FDA has unveiled draft guidance designed to assist rare disease therapy developers by providing regulatory clarity in…
Children, adolescents see Spinraza benefits in real-world study Written by Andrea Lobo | Oct. 26, 2026 Treatment with Spinraza (nusinersen) led to sustained improvements in motor function in children and adolescents with spinal muscular atrophy (SMA), according to a real-world study from Czechia…
By Emily Leclerc, Waisman Science Writer It’s hard to predict how a small decision may make a major impact on a person’s life. For Albee Messing, VMD, PhD, Waisman investigator, the decision to pursue what others deemed a ridiculous idea…
The ‘heart’ of the Fanconi anemia core complex, composed of FANCB-FANCL-FAAP100 (now FANCX) proteins, allows the rest of the core complex to assemble, and is necessary for downstream DNA repair. From patient to pathway It was then that FAAP100 officially gained…
Dublin, Oct. 29, 2026 (GLOBE NEWSWIRE) -- "Shotgun Metagenomics Clinical Reporting Market Global Report 2026" has been added to ResearchAndMarkets.com's offering. The global shotgun metagenomics clinical reporting market is experiencing rapid growth as healthcare organizations increase their use of advanced genomic testing for…
Cell and Gene Therapies (CGTs) present unique development challenges, from novel manufacturing processes and delivery methods to complex nonclinical programs and evolving regulatory expectations. For sponsors developing these therapies, engaging with the FDA early and often can help clarify development…
Working with clinical researchers at Cleveland Clinic Abu Dhabi, the brain-computer interface company takes next step toward bringing transformative neurotechnology to patients worldwide... Precision Neuroscience Expands Clinical Network Beyond the U.S. with First International Site in Abu Dhabi Working with clinical…
BioMarin will make an upfront payment of $70 million; GC and Hanmi will be jointly eligible to receive additional payments upon certain development and commercial milestones, and tiered royalties on net sales of HM15421/GC1134A SAN RAFAEL, Calif., Oct. 29, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc.
Our experienced, multidisciplinary team of specialists provides comprehensive care for all forms of NF and supports the emotional well-being of patients and fam Genetic Testing and Genetic Counseling for Neurofibromatosis Type 1 (NF1) In most cases, a diagnosis of NF1 is made through…
Rare diseases in general: long searches for a diagnosis, orphan drugs, Rare Disease Day and patient advocacy.
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