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Biomedical subjects

M Super

Publications and source records attributed to M Super.

119 records · Page 7Linked to original sources

Cystic fibrosis in the South West African Afrikaner. An example of population drift, possibly with heterozygote advantage.

An incidence of the genetic recessive disease cystic fibrosis (mucoviscidosis) far in excess of that reported recently from other countries, has been encountered in the South West African Afrikaner. This has probably resulted from the immigration of a segment of the South African Afrikaner population rich in the gene, into South West Africa, where, for religious reasons and reasons of geographical isolation, the gene has persisted and, perhaps, increased in frequency. Malaria, which killed many of the early settlers, might have selectively spared carriers of the gene, thus enriching its occurrence in the population. Details of patients, particularly with regard to the criteria of diagnosis, are given with the relevant population and birth figures, from which an estimate of the incidence of the disease and of its carrier rate has been made. Screening of the newborn for the condition and the compiling of a register of potential and obligatory carriers are also discussed.

Africa, Southern↗

Phocomelia.

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Abnormalities, Drug-Induced↗

Amplification refractory mutation system for prenatal diagnosis and carrier assessment in cystic fibrosis.

The amplification refractory mutation system (ARMS) has been applied to prenatal diagnosis and carrier detection of cystic fibrosis. The nucleotide sequence of both alleles of the PstI restriction fragment length polymorphism at the KM19 locus, which displays linkage disequilibrium with cystic fibrosis, has been determined. ARMS enables direct analysis of alleles of this polymorphism in DNA isolated from chorionic villus biopsy or white blood cells.

Alleles↗

Gonadotrophin-independent precocious puberty and dyschondrosteosis.

We present a case of gonadotrophin-independent precocious puberty, as seen in McCune-Albright syndrome, and Leri-Weill dyschondrosteosis in a six and a half year-old girl. Her father also reports having early puberty and is similarly affected with dyschondrosteosis. This combination of features has not been reported previously, and represents the association of a skeletal dysplasia with an endocrinopathy.

Bone Diseases, Developmental↗

Genetic advances in cystic fibrosis: to screen, to treat or both?

Since the identification of the cystic fibrosis (CF) gene in 1989 there have been considerable advances in treatment and fierce debate concerning CF screening programmes. Cystic fibrosis imposes a burden of suffering, of onerous treatment and of reduced lifespan. New emerging treatments are very expensive but are expected to improve survival and quality of life. Treatments and screening programmes should not be played off against one another in choices for funding. This paper presents an argument for the appropriate use of newer treatments which could be funded from savings arising from the effects of widespread implementation of newborn and active cascade carrier detection programmes. Newborn screening delays the need for expensive treatments reserved for those with advanced disease, by reducing pre-diagnosis tissue damage and the rate of progression of the disease. Carrier screening has the capacity to decrease the numerical burden of affected children born and increases the number of children born free of disease. For the present, carrier screening in the absence of a family history could be funded by the individuals who wish it or by those prepared to buy this service. All types of screening presume quality control, an existing laboratory and counselling infrastructure and an established method of communicating results.

Cost of Illness↗