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Mitochondrial Disorders
CONCLUSION: Patients with mitochondrial diseases have highly varied manifestations and can thus present to physicians in practically any branch of medicine. A correct diagnosis is the prerequisite for genetic counseling and for the initiation of personalized treatment.PMID:34158150 | DOI:10.3238/arztebl.m2021.0251
Source: Deutsches Arzteblatt International - June 23, 2021 Category: General Medicine Authors: Thomas Klopstock Claudia Priglinger Ali Yilmaz Cornelia Kornblum Felix Distelmaier Holger Prokisch Source Type: research

Gene Therapy for Monogenic Inherited Disorders
CONCLUSION: The first gene treatments have now been approved and bring hope of long-term therapeutic benefit after a single administration. The numbers of patients who come into question for specific therapies are often low, so that many different aspects- generation of evidence on efficacy and safety, determining indications, performance of the treatment, pricing-bring new challenges.PMID:33637169 | DOI:10.3238/arztebl.2020.0878
Source: Deutsches Arzteblatt International - February 27, 2021 Category: General Medicine Authors: Janbernd Kirschner Toni Cathomen Source Type: research

Treatment Options in Hemophilia.
CONCLUSION: Now that new alternatives to classic supplementation therapy are be- coming available, pertinent treatment algorithms for patients with hemophilia will have to be developed. It is still unclear to what extent the new drugs might supplant clotting factor supplementation as the first line of treatment. PMID: 31847949 [PubMed - in process]
Source: Deutsches Arzteblatt International - December 20, 2019 Category: General Medicine Tags: Dtsch Arztebl Int Source Type: research