Sentences with phrase «affected by rare diseases»

Rare Revolution Magazine is a UK - based digital magazine giving a voice to patients affected by rare diseases and the charities that represent and support them.

The specific vocation of I - Stem is to explore all the therapeutic potential of human pluripotent stem cells for applications in patients affected by rare diseases of genetic origin.

The goal of CARE for RARE is to improve clinical care for patients and families affected by rare diseases by expanding and improving the diagnosis and treatment of rare diseases.

Chiesi Ventures is unique in its focus: improving quality of diagnosis, disease management, care and therapy to all patients affected by rare diseases.

Collectively, an estimated 30 million people are affected by a rare disease in Europe with a similar occurrence in the US.

Approximately one out of 20,000 newborns is affected by this rare disease which is considered to be one of the most severe forms of malformations on this spectrum.

Not exact matches

Rare diseases are conditions affecting less than 200,000 people in the US and less than five (5) in 10,000 according to the definition adopted by the European Medicines Agency.

Cystic fibrosis is a rare inherited disease caused by mutations in a single gene called cystic fibrosis transmembrane conductance regulator (CFTR) and affects 1 in every 2500 newborns in the UK and over 90000 people worldwide.

DMD is a rare disease affecting primarily boys and is caused by defects in the gene that makes the dystrophin protein.

This rare and deadly disease mainly affects young women, and is characterized by very high pressure in the arteries supplying blood to the lungs.

Anna Gunby can't run around as smoothly as most 4 - year - olds because her wobbly legs are affected by a rare brain disease that also hinders her intellect.

Collaborative Group of the Americas on Inherited Colorectal Cancer aims to advance research on several rare forms of inherited colorectal cancers, as well as improve clinical care for patients and families affected by these diseases.

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults.

We are passionate about educating and supporting patients, families and caregivers affected by rare and ultra-rare diseases.

The UK 100,000 Genomes Project is a UK large - scale genomic project aiming to sequence and analyse the genetic information of NHS patients affected by cancers or rare diseases.

Sarepta Therapeutics formerly AVI BioPharma remains focused on developing RNA - based therapeutics to improve and save the lives of people affected by serious and life - threatening rare and infectious diseases.

If, on closer examination, it becomes apparent that patients with rare diseases are disproportionately denied funding for medical services or disproportionately affected by denial of funding, then differential treatment is established.

AGTC's lead product candidates focus on rare diseases of the eye, caused by mutations in single genes, that significantly affect visual function and currently lack effective medical treatments.

One person who has been helped by Friendship Place is 56 - year - old Anthony Silas, who was homeless for six years after a rare disease affecting his brain and spinal cord caused him to lose his job.