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UK lags behind on access to treatments for rare diseases

Drug Discovery World

The UK lags comparable countries, including France and Germany, on the degree of availability of treatments for rare diseases, according to a new report. In response, the BioIndustry Association (BIA)’s Rare Disease Industry Group (RDIG) is calling for the UK to improve access to new treatments for patients.

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Which diseases are poised to benefit from therapeutic antibody development

Drug Discovery World

DDW’s Megan Thomas looks at how different diseases will benefit from the success of therapeutic antibodies. billion in 2021, and is projected to reach $106.8 What is particularly noteworthy when it comes to the mAbs being developed and approved for Covid-19 is drug repurposing, showing the scope of future treatments with mAbs.

Disease 147
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Antibody treatment for geographic atrophy enters clinical trials

Drug Discovery World

Collaborators Boehringer Ingelheim and CDR-Life have commenced a Phase I evaluation of BI 771716, their antibody fragment-based treatment developed to preserve the vision of people with geographic atrophy (GA). GA is an irreversible retinal disease that occurs in people with late-stage dry age-related macular degeneration (AMD).

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European coalition to accelerate research into 7000 rare diseases

Drug Discovery World

The ‘Rare Disease Moonshot’, a new initiative to boost research and development into rare and paediatric diseases, has launched at the European Health Summit. . It aims to speed up research into more than 7000 identified rare diseases, of which a staggering 95% have no approved treatment or cure. .

Disease 130
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Rhythm Pharmaceuticals buys biotech focused on rare disease

Drug Discovery World

CHI is a rare genetic disease in which cells secrete excess insulin, causing hypoglycemia, which can result in serious health outcomes including seizures, coma, permanent brain damage and death. Xinvento is developing novel investigational therapeutic candidates designed to improve the care of patients with CHI.

Disease 130
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SIGA’s monkeypox treatment gets UK approval  

Drug Discovery World

Pharmaceutical company SIGA Technologies has announced that its oral therapy tecovirimat has been approved in the UK for the treatment of smallpox, monkeypox, cowpox. . The therapy has also been approved for the treatment of complications following vaccination against smallpox in adults and children with a body weight of at least 13kg. .

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FDA Fast Track Designation for retinitis pigmentosa treatment

Drug Discovery World

The drug was granted orphan drug designation by the FDA in May 2021. RP consists of a group of inherited diseases causing slow and progressive retinal degeneration and loss of vision, for which there is currently no treatment for most patients. It is a leading cause of inherited blindness, with an estimated 1.5

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