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Also known as:Lubani-Al Saleh-Teebi syndromecystic fibrosis gastritis megaloblastic anaemiacystic fibrosis gastritis megaloblastic anemia
Overview
A rare genetic disease reported in two siblings of consanguineous Arab parents and is characterized by cystic fibrosis, gastritis associated with Helicobacter pylori, folate deficiency megaloblastic anemia, and intellectual disability. There have been no further descriptions in the literature since 1991.
Diagnosis
Biomarker and diagnostic research for cystic fibrosis-gastritis-megaloblastic anemia syndrome has been reported in the published literature.
Prognosis
Estimated prevalence: <1 in 1,000,000 (VERY_RARE).
Research Landscape
Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.
No clinical trials have been registered for cystic fibrosis-gastritis-megaloblastic anemia syndrome.
123 publications have been identified in PubMed for cystic fibrosis-gastritis-megaloblastic anemia syndrome. Research spans Review / Meta-Analysis (64%), Basic Science / Preclinical (14%), and Case Report / Case Series (11%).
Research Type
Count
% of Total
Research summaries
72
64%
Data assembled from 4 of 12 sources · Last updated Nov 5, 2026, 4:28 AM UTC
Diseases are connected by shared genes, phenotypes, therapeutic targets, and MONDO disease hierarchy. The Rare badge reflects Kisho's rarity classification — scored from authoritative sources including OMIM, Orphanet, orphan drug designations, clinical trial activity, and published prevalence data.
What investors expect at each round, how much companies typically raise, which non-dilutive money to pursue, and how to read a term sheet before you sign one. Rather than keep raising money, the company is bought by a pharmaceutical or biotech company looking to add a drug candidate, expand its pipeline, or enter a new therapeutic area. An IPO raises capital while the company stays independent. An acquisition transfers ownership and control to the buyer. Venture philanthropy: Disease foundations and patient groups fund programs aligned with their missions, through grants, loans, or equity. The best-known case is the Cystic Fibrosis Foundation, which put $150 million into Vertex Pharmaceuticals' cystic fibrosis program in exchange for royalties. In 2014, it sold those royalty rights to Royalty Pharma for $3.3 billion. Partnerships and licensing deals: Upfront and milestone payments from pharma partners are a major source of non-dilutive capital, especially for platform companies that can license one asset or application while keeping others. Turning a promising scientific discovery into a biotech company takes more than good science. It takes money to move an idea from the lab to the clinic, build a team, navigate regulatory requirements, develop a payer strategy, and ultimately bring new treatments to the people who need them. Raising that money is rarely linear. Public investors price them on clinical data, the market for their lead programs, and the cash runway to the next readout. The bar has risen since the 2021 boom. Of the six biotechs that priced IPOs in the first quarter of 2026, all but one had drugs in mid- or late-stage testing, and no preclinical company had gone public since 2024, according to BioPharma Dive.
ReCode Therapeutics appoints a new CEO to lead its efforts in advancing clinical programs for cystic fibrosis and primary ciliary dyskinesia. This leadership change follows the company's recent clinical validation earlier this year.
A recent study demonstrates the efficacy of the combination therapy Elexacaftor/Tezacaftor/Ivacaftor in cystic fibrosis patients with severe lung disease who carry previously ineligible CFTR mutations. This finding could expand treatment options for a broader patient population.
