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Sasha Stolt

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Apr 18, 2024, 3:33:55 PM4/18/24
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We are CURE Epilepsy - the leading nongovernmental agency fully committed to funding research in epilepsy.

Our organization was founded by Susan Axelrod and mothers of children with epilepsy who joined forces to spearhead the search for a cure.

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The Cure Sickle Cell Initiative is an NHLBI-led collaborative research effort that is accelerating the development of gene therapies to cure sickle cell disease. The Initiative identifies and supports promising gene therapies that are currently being tested in multicenter clinical research trials. One of these studies is co-funded by the California Institute of Regenerative Medicine (CIRM). Preliminary results of these clinical trials are expected within three to five years. The Initiative aims to transform the lives of people who have sickle cell disease by creating a collaborative, patient-focused research environment. With new advancements in gene therapy, the time is right to push toward cures that can be offered to many of the approximately 100,000 Americans, and more than 20 million people worldwide, who have sickle cell disease.

Currently, bone marrow transplants can cure sickle cell disease, but they are most effective in children who have well-matched donors. The Cure Sickle Cell Initiative aims to develop curative strategies by initially focusing on gene therapies that modify hematopoietic stem cells (HSCs), which make red and other blood cells. These modified HSCs can then be used in gene therapy, making a cure available to more patients.

Given the limitations of antiretroviral therapy and recent advances in our understanding of HIV persistence during effective treatment, there is a growing recognition that a cure for HIV infection is both needed and feasible. The International AIDS Society convened a group of international experts to develop a scientific strategy for research towards an HIV cure. Several priorities for basic, translational and clinical research were identified. This Opinion article summarizes the group's recommended key goals for the international community.

While there is currently no cure for HIV, advances in treatment have made it possible for people with HIV to live long and healthy lives. We also now have more tools to halt the epidemic through treatment, prevention and education. Still, a cure would facilitate the global eradication of HIV/AIDS. For this reason, NIAID invests in basic and clinical research aimed at developing a safe, affordable and scalable cure for HIV and AIDS. Watch a video to learn about the approaches NIAID is taking to achieve a cure for HIV.

Many people involved in HIV cure research acknowledge that, much like the best treatments for HIV, a cure may consist of a combination of agents and approaches. Because of the nature of HIV infection, a cure for HIV can be defined in two ways: treatment-free remission and viral eradication.

Many people living with HIV who adhere to regular antiretroviral therapy (ART) maintain undetectable levels of the virus in their blood. While these individuals have effectively no risk of sexually transmitting HIV and are less likely to experience most symptoms and complications of HIV infection, latent virus remains in certain cells known collectively as the HIV reservoir. If people with ART-suppressed HIV stop taking their medication, virus from the reservoir rebounds to high levels. Sustained ART-free remission, sometimes called a functional cure, would allow a person living with HIV to keep latent virus suppressed without daily medication. Read more about NIAID-supported research toward sustained ART-free remission.

A plan administrator may, but is not required to, allow a cure period during which a delinquent participant loan may be brought back into compliance without triggering a deemed distribution. If allowed, the cure period must be specifically provided for in the written plan document.

If this option is adopted, the maximum allowable cure period would extend to the last day of the calendar quarter following the calendar quarter in which the required installment payment was due. Reg. Section 1.72(p)-1, Q&A-10(a). The plan administrator may also adopt a shorter cure period, or none.

If a loan repayment is still late even after considering this cure period, then the amount of the deemed distribution is equal to the entire outstanding balance of the loan (including any accrued interest) as of the last day of the cure period. Reg. Section 1.72(p)-1, Q&A-10(b).

CCA 201736022 describes two ways in which missed installment payments can be made up during the cure period. One involves making up the missed payments during the cure period. The other involves refinancing the existing loan, also within the cure period.

In this example, the make-up installment payment was received after the end of the cure period for the March 31 installment. As a result, Participant A has a deemed distribution on June 30. The amount of the deemed distribution is the outstanding loan balance (including accrued interest) on June 30. However, because the participant made loan repayments after the deemed distribution occurred on June 30, the participant has an investment in the contract (tax basis) equal to the amount of the payment made on July 3. See Reg. Section 1.72(p)-1, Q&A-21.

The missed payments are in the fourth calendar quarter and have the same cure period ending March 31, Year 3. Each missed installment is cured by the timely refinancing of the loan prior to the end of the cure period.

Informational bulletins, which include answers to frequently asked questions, are available at the Department of Justice CURES Program website. CURES users are encouraged to monitor the website for additional information and instructions prior to the implementation of this change. Please direct questions regarding these updates to the CURES Program at cu...@doj.ca.gov or (916) 210-3187.

In December 2023, the Cure CRC Summit welcomed more than 100 prominent scientists to foster partnerships that will challenge the status quo of colorectal therapies and discover new trajectories toward a cure.

People with hepatitis C may also benefit from lifestyle changes, such as avoiding alcohol and maintaining a healthy weight. With proper treatment, many people can be cured from hepatitis C infection and live healthy lives.

WHO recommends therapy with pan-genotypic direct-acting antivirals (DAAs) for all adults, adolescents and children down to 3 years of age with chronic hepatitis C infection. The short-course oral, curative DAA treatment regimens has few if any side-effects. DAAs can cure most persons with HCV infection, and treatment duration is short (usually 12 to 24 weeks), depending on the absence or presence of cirrhosis. In 2022, WHO included new recommendations for treatment of adolescents and children using the same pangenotypic treatments used for adults.

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It is safe to say that RLS is a real clinical entity composed of clearly recognizable symptoms, with no cure and no ending, unless it is associated with iron-deficiency anemia. However, as a disease, it seems to lack etiology, pathology, pathogenesis, pathophysiology, diagnostic findings on physical examination, laboratory tests, or imaging, and any clear strategy for prevention.

The National Institutes of Health announced the launch of a new initiative to help speed the development of cures for sickle cell disease, a group of inherited blood disorders affecting at least 100,000 people in the United States and 20 million worldwide. The Cure Sickle Cell Initiative will take advantage of the latest genetic discoveries and technological advances to move the most promising genetic-based curative therapies safely into clinical trials within five to 10 years.

Decades of basic research on sickle cell disease have laid the groundwork for novel genetic approaches to cures, such as the genetic editing of bone marrow cells, which have shown great promise in animal models and in some small scale human studies. In addition, the NHLBI Production Assistance for Cellular Therapies (PACT) program has been working with researchers to manufacture cellular therapeutic products, including genetically modified cells, that can be used safely in clinical trials with patients.

Currently, the only cure for sickle cell disease is a bone marrow transplant, a procedure in which a sick patient receives bone marrow from a healthy, genetically-compatible sibling donor. However, transplants are too risky for many adults, and only about 18 percent of children with sickle cell disease have a healthy, matched sibling donor.

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