Afleveringen
-
It's a thin week for acromegaly-specific research — just one mechanistic study on tumor biology — but rare disease dealmaking was busy, headlined by Ipsen's ~$800M bet on a kidney-transplant antiviral and an FDA age expansion for Vertex's CRISPR therapy Casgevy.
-
Paltusotine's four-year open-label data confirm durable oral control of acromegaly in patients switched from injections, while the rare disease industry absorbs a landmark Sangamo bankruptcy, a well-funded gene-editing entrant in AATD, and a new build-to-buy cardiomyopathy venture from Merck KGaA.
-
Zijn er afleveringen die ontbreken?
-
A rich week in acromegaly research — new data on renal complications, a precision medicine framework, a high-risk co-secreting tumor phenotype, and cancer risk analyses — while the rare disease industry continues to see a wave of FDA regulatory reversals benefiting gene therapy developers.
-
Surgical technique innovation and a JCEM editorial from two giants of the field reshape how we think about curing GH-secreting tumors and managing cancer risk in acromegaly, while the rare disease sector sees a burst of late-stage deal-making and platform financing that signals strong investor conviction in gene-silencing technologies.
-
A landmark JCEM special issue lands this week with five major reviews and original studies that collectively reset the clinical playbook for acromegaly — from emerging oral therapies to the underappreciated mortality burden — while in the rare disease industry, Alnylam bets $2 billion on AI-designed RNA medicines, a controversial FDA voucher program faces calls for reform, and a new market forecast projects orphan drug sales above $400 billion by 2032.
-
New Pituitary research this week highlights the cardiac and bone risks that persist even in treated acromegaly patients, while the rare disease industry grapples with a mixed regulatory environment — one notable ASCO data release and continued fallout from FDA leadership turnover.
-
A network meta-analysis confirms that injectable somatostatin receptor ligands remain first-line in acromegaly, while new clinical data on paltusotine and pasireotide, plus rare disease industry news including BioMarin and Intellia, shape the week.
-
New proteomic biomarkers could sharpen acromegaly diagnosis beyond GH/IGF-1, while the rare disease industry sees a wave of FDA decisions and high-stakes M&A activity.
-
Acromegaly research this week centered on the long-term pharmacology of established therapies and clinical management updates, while the rare disease industry absorbed significant regulatory and pipeline news.
-
This week's literature brings a long-term safety confirmation for pegvisomant monotherapy, a head-to-head comparison of first-generation somatostatin analogs using a validated composite score, and new evidence linking acromegaly to hepatic steatosis — alongside cost-effectiveness data for pasireotide and updated pituitary tumor pathology tools.
-
Two important consensus-level papers from late April 2026 sharpen the standards for acromegaly diagnosis and comorbidity management, incorporating AI-assisted screening, ultrasensitive GH assays, and updated international expert guidance.
-
This week's acromegaly literature is headlined by a landmark cardiovascular disease review in Endocrine Reviews and a cluster of high-yield JCEM papers covering biomarker-guided therapy, standardized pituitary imaging, patient-reported quality of life, and histological predictors of surgical remission.
