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Rare disease spotlight: treating Friedreich's ataxia

Rare disease spotlight: treating Friedreich's ataxia
europeanpharmaceuticalreview.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from europeanpharmaceuticalreview.com Daily Mail and Mail on Sunday newspapers.

Nguengang Wakap , Sylvia Boesch , Toby Ferguson , University Of Pennsylvania , European Commission , Rare Movement Disorders Innsbruck , Head Of The Center , Head Of The Neuromuscular Development Unit , Committee For Medicinal Products Human Use , Department Of Neurology , University Of Florida , University Innsbruck , Reata Pharmaceuticals , Vice President , Neuromuscular Development Unit , Medicinal Products , Human Use , Rare Disease Day , Principal Investigator , Medical University Innsbruck , Friedreich Ataxia Rating Scale , Phd Toby , Eurj Med ,

Contributor: How Patients and Caregivers Can Be a Catalyst for Rare Disease Innovation

Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence regulators and payers' decisions, ultimately serving as catalysts for innovation in the field. ....

United States , Kyle Bryant , Philip Cyr , Nguengang Wakap , International Meeting , Drug Administration , Friedreich Ataxia Research Alliance , Agency For Healthcare Research , Ataxia Research Alliance , Orphan Drug Act , Drug Evaluation , Innov Regul , Annual International , Annual European Meeting , Accessed September , Evaluating Patient Outcomes , Healthcare Research , Rare Disease , Future Rare , Health Access ,

From target discovery to clinical drug development with human genetics

The substantial investments in human genetics and genomics made over the past three decades were anticipated to result in many innovative therapies. Here we investigate the extent to which these expectations have been met, excluding cancer treatments. In our search, we identified 40 germline genetic observations that led directly to new targets and subsequently to novel approved therapies for 36 rare and 4 common conditions. The median time between genetic target discovery and drug approval was 25 years. Most of the genetically driven therapies for rare diseases compensate for disease-causing loss-of-function mutations. The therapies approved for common conditions are all inhibitors designed to pharmacologically mimic the natural, disease-protective effects of rare loss-of-function variants. Large biobank-based genetic studies have the power to identify and validate a large number of new drug targets. Genetics can also assist in the clinical development phase of drugs—for example ....

San Francisco , United States , Southwest Finland , Van Goor , Lopes Alves , Davey Smith , Nguengang Wakap , Finasteride Male Pattern Hair Loss Study Group , Finasteride Study Group , Precision Medicine Initiative All Of Us , Health Drug , Estonian Genome Center , American Heart Association , Collaborative Group , Human Services , Us Department Of Health , Disease Diagnosis In Health Care , University Of Tartu , Wellcome Trust Case Control Consortium , Africa Consortium , Development Study , Regeneron Genetics Center , European Medicines Agency , Genomics Beyond Health Government Office For Science , Drug Discov , Human Genome ,

The Need for and Impact of Rare Disease Education

- PMLiVE ....

Nguengang Wakap , National Rare Disease , Intj Environ Res Public Health , Rare Disease , Medscape Education , Rare Disease Survey , Intj Environ Res Public ,

We are many: Advocating for patients with rare diseases

We are many: Advocating for patients with rare diseases
georgeherald.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from georgeherald.com Daily Mail and Mail on Sunday newspapers.

South Africa , South Africans , Monique Nel , Nguengang Wakap , Rare Humanitarian Programme , Rare Disease Information Center , National Treasury , Sanofi Your Health , Sanofi Genzyme Rare Humanitarian Program , Disease Day , Rare Diseases , Rare Disease Policy , Medical Advisor , Rare Disease Information , About Rare , Accessed January , Genet Metab , Accessed February ,