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Slow Rollout of First CRISPR-Based Treatment for Sickle Cell Disease

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Mon 23rd Dec, 2024

Deshawn Chow, a 19-year-old diagnosed with sickle cell disease, recently received a groundbreaking treatment known as Casgevy, which is the first approved gene-editing drug utilizing CRISPR technology. This innovative therapy has the potential to alleviate the severe pain and complications associated with his condition, which affects approximately 100,000 individuals in the United States, predominantly among the Black community.

Chow’s journey to treatment began a year ago when he learned about Casgevy, which was officially approved in December 2023 for sickle cell disease and beta thalassemia. After enduring frequent hospital visits and debilitating pain episodes throughout high school, Chow finally received the therapy on December 5 at the City of Hope Cancer Center in Los Angeles. However, the rollout of Casgevy has been slow due to various factors, including manufacturing complexities and insurance challenges.

The process of administering Casgevy is intricate and not as straightforward as traditional medications. Patients undergo a preparatory phase that involves chemotherapy to create space in the bone marrow for the edited stem cells. Chow’s hematologist emphasized that the treatment requires a significant commitment, including a month-long hospital stay following the stem cell infusion.

Despite its promise, only a limited number of patients have been treated with Casgevy since its approval. Hospitals across the country are navigating the complexities of the therapy’s administration and insurance approval processes. Of the 34 U.S. hospitals authorized to provide Casgevy, many have yet to begin treatment due to logistical hurdles, with only a few patients receiving the drug so far.

The clinical trials preceding Casgevy’s approval demonstrated significant success, with most participants experiencing relief from pain crises for extended periods after treatment. The average duration without pain crises reported was over two years, with some individuals remaining pain-free for up to five years. This success has sparked considerable interest among patients and healthcare professionals alike, indicating a potential shift in the management of sickle cell disease.

However, there are concerns regarding the technology’s reception, particularly among Black patients who have historically faced distrust in the medical system. The lengthy treatment process, which resembles a stem cell transplant involving multiple steps and potential side effects, can deter patients who are not fully informed about the procedure.

Chow and his family proactively sought information about Casgevy after its approval, understanding the risks and benefits involved. While the treatment offers hope, it also requires navigating a complex healthcare landscape, including lengthy insurance approval processes and substantial out-of-pocket costs. The drug itself is priced at $2.2 million, which does not account for the extensive medical care leading up to and following the treatment.

As hospitals continue to build their infrastructure to offer this cutting-edge therapy more efficiently, experts anticipate a gradual increase in patient demand. The key will be to address the existing barriers to access and improve patient education about the treatment process. With ongoing advancements, there is hope that Casgevy could revolutionize the standard of care for sickle cell disease, paving the way for a future where pain management is significantly improved for those affected.

Chow remains hopeful about the outcome of his treatment. He envisions a future where he can engage in activities that were previously hindered by his condition, such as snowboarding and spending time with friends. As he recuperates in the hospital, he continues to pursue his passion for music, reflecting on the journey that has brought him to this point.

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Dortmunderin (85) gab entscheidenden Hinweis

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Dortmunderin (85) gab entscheidenden Hinweis

In Dortmund ist eine Betrugsmasche rund um die Pflege von Senioren aufgeflogen. (Symbolbild) © picture alliance / Jens Kalaene/dpa-Zentralbild/dpa

Lesezeit

Eine 85-jährige Dortmunderin aus Wickede hat durch ihre Aufmerksamkeit dazu beigetragen, dass ein umfangreicher Pflegebetrug aufgedeckt und ein finanzieller Schaden von über 500.000 Euro verhindert werden konnte. Das berichtet die Polizei Dortmund.

Die Seniorin habe das Ausbleiben der Zahlungen ihres Pflegegeldes bemerkt und daraufhin die Beamten informiert. Der Verdacht fiel auf ihren vermeintlichen Pflegehelfer, den ein 77-jähriger Bekannter vermittelt hatte, und sich schließlich als Teil eines umfassenden Betrugskonstrukts herausstellte.

Es sei gelungen, drei Tatverdächtige zu identifizieren. Infolgedessen wurden Durchsuchungen durchgeführt und Beweismittel sichergestellt.

In einem Gebäude auf einem landwirtschaftlichen Gelände in Dortmund ist ein Feuer ausgebrochen.
Junge Frau mit verpixeltem Gesicht sitzt auf Erdbeerfeld. Neben sich eine Schrift ins Bild geschnitten: „Illegal auf dem Erdbeerfeld“

Wie der Pflegebetrug ablief

Die Ermittlungen zeigten, dass einer der Verdächtigen das Vertrauen des 77-jährigen Bekannten der Seniorin über Jahre missbraucht und ihm mehr als 25.000 Euro entwendet haben soll. Darüber hinaus habe der Verdächtige versucht, mittels Vollmachten und eines Testaments Zugriff auf dessen Vermögen zu erlangen.

Gegen alle Verdächtigen, zwei Männer im Alter von 26 und 34 Jahren sowie eine 42-jährige Frau, seien Strafverfahren wegen Betrugs zum Nachteil älterer Menschen eingeleitet worden. Allerdings lagen keine besonderen Voraussetzungen für eine Untersuchungshaft vor.

Eine Außenaufnahme eines Polizeireviers an einem sonnigen Tag. Im Vordergrund ist der Eingang des Gebäudes zu sehen, während im Hintergrund ein Streifenwagen geparkt ist.

Tipps für Angehörige

Um pflegebedürftige Menschen vor ähnlichen Betrugsfällen zu schützen, gibt die Polizei mehrere Präventionstipps. Es wird geraten, keine Unbekannten in die Wohnung zu lassen und stets einen Dienstausweis zu verlangen, wenn sich Personen als Amtspersonen oder Beschäftigte von Pflegediensten ausgeben. Sollte Zweifel an der Identität bestehen, sei es ratsam, die betreffende Stelle direkt zu kontaktieren. Telefonische Anfragen sollten kritisch geprüft werden, ohne private oder finanzielle Informationen preiszugeben, und bei Unsicherheit solle einfach aufgelegt werden.

Von der Übergabe von Geld oder Wertgegenständen an Unbekannte wird ausdrücklich abgeraten. Auch sei es wichtig, auf das eigene Bauchgefühl zu vertrauen. Im Verdachtsfall solle die Polizei unter der Notrufnummer 110 informiert werden.

Darüber hinaus bietet die Polizei spezielle Präventionsveranstaltungen und Beratungen für Senioren und deren Angehörige an, um über gängige Betrugsmaschen aufzuklären und Schutzmaßnahmen zu vermitteln. Interessierte können sich auf der Homepage der Polizei weiter informieren.

Diese Maßnahmen sollen dazu beitragen, dass pflegebedürftige Menschen und ihre Angehörigen besser vor betrügerischen Machenschaften geschützt werden und die Täter keinen Erfolg mit ihren kriminellen Handlungen haben.

Hinweis der Redaktion: Dieser Artikel erschien ursprünglich am 28. Juli 2025.

Der Angeklagte sitzt mit seinem Verteidiger Markus Blumenstein im Gerichtsaal.

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Tragic Capsizing of Overloaded Ferry Claims Lives in DR Congo

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Sun 22nd Dec, 2024

A devastating incident occurred in the Democratic Republic of the Congo when an overloaded ferry capsized on a river, leading to a significant loss of life. Reports indicate that the vessel was carrying many more passengers and cargo than it was designed to accommodate, raising serious concerns about maritime safety standards in the region.

Emergency services and local authorities were quick to respond to the scene, but the rescue operations faced severe challenges due to the conditions of the river and the high number of people involved. Eyewitness accounts suggest that panic ensued as the ferry began to tilt and eventually overturned, throwing passengers into the water.

Survivors described harrowing scenes of chaos, with many struggling to stay afloat amidst the turbulent waters. The local community has rallied together to assist in rescue efforts, providing support to those affected and helping search for those still missing.

This tragic event highlights ongoing issues related to safety regulations in the region’s transportation sector. Overcrowding on ferries is not uncommon in the Congo, where many rely on these vessels for travel across rivers, which are vital for connecting remote communities. However, this incident raises urgent questions about the enforcement of safety protocols and the need for stricter regulations to prevent similar occurrences in the future.

Authorities have begun an investigation into the circumstances surrounding the capsizing. Initial reports suggest that the ferry’s operators may have disregarded safety guidelines, leading to the tragic overloading of the vessel. As the investigation unfolds, officials are expected to review existing regulations and operational practices within the ferry transport sector.

In the wake of the accident, various organizations and governmental bodies are calling for enhanced safety measures to protect passengers. There is a growing acknowledgment that without significant reforms, such tragedies could continue to occur, jeopardizing the lives of countless individuals who depend on river transport.

The humanitarian response is underway, with local groups providing assistance to families affected by the loss of loved ones. Mental health support and counseling services are also being offered to help individuals cope with the trauma of this disaster.

As the community mourns the victims of this incident, there is a collective hope that this tragedy will serve as a catalyst for change. Advocates for transport safety are urging policymakers to take immediate action to improve safety standards in the ferry sector, ensuring that such a loss of life is not repeated.

This incident serves as a stark reminder of the vulnerabilities faced by those who utilize river transport in the Democratic Republic of the Congo and underscores the urgent need for systemic changes to safeguard the lives of passengers.

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German Chancellor Merz welcomes EU trade deal with US

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German Chancellor Merz welcomes EU trade deal with US

German Chancellor Friedrich Merz on Sunday welcomed the trade deal between the United States and the European Union, which he said avoided ‘needless escalation in transatlantic trade relations’ — even as many industries criticised it.

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