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Kansas City family worries cost could keep drug from helping son with rare disorder

A family in Kansas City, Missouri, hopes that existing medications can help his son’s rare illness. However, it costs more than $ 700,000 a year, so I’m worried that it may be out of reach. “Charlie was a rough few months,” said Kim Fry. Fry said her life was consumed by finding relief for her 3-year-old son suffering from a rare genetic disorder. “95% of rare diseases, including Charlie, do not have an approved treatment,” she said. This disorder is called SLC6A1 and is named after the affected gene. It causes seizures and delayed development. “He’s taking epilepsy medication and we have a lot of really difficult side effects,” Fry said. As she and other families raise money to fund gene replacement research, another drug is promising. “SLC6A1Connect has funded a research study to test approved FDA drugs to see if they can hit anything that can help children. Ravicti returns as a really powerful option. I’ve done it, “says Fry. “We were very excited and brought great hope to the whole family that we could get a safe FDA-approved drug with few side effects.” Ravicti is manufactured by Horizon Therapeutics. -A week-long study of children with seizure disorders such as SLC6A1. The drug price tag made Fly worried about what would happen at the end of the test. “It’s $ 740,000 a year, more than $ 2,000 a day,” she said. “It’s not just an option.” Insurance doesn’t cover it now because it hasn’t been approved yet due to Charlie’s disability. “Knowing that there is something that might help Charlie is really devastating and emotional, and I don’t have access to it,” Fry said. She said the company wants to work with families like her to make the drug more affordable. “We want our children to be healthy and happy and have the same chances of life as everyone else,” she said. Horizon Therapeutics said it was waiting for the results of the trial before deciding whether to expand access to the drug. In a statement to KMBC 9, Horizon said: We worked closely with the investigator to confirm that there are routes for investigators to request treatment beyond the trial period. It is based on the investigator’s clinical evaluation and oversight by the Institutional Review Board (IRB). Horizon has not denied anyone access to our drug in this trial. “Horizon is a company that is deeply involved with people with rare diseases. We have a long track record of doing the right thing and giving patients access to our medicines. Horizon’s Deputy Communications and Patient Support Officer. President Matt Fresh told KM BC 9 on the phone: “Currently, less than 1,000 people in the United States are receiving Ravich for the FDA.-Approved Indications (Urea cycle disorder). This is an incredibly small patient population. Therefore, it is priced as a drug for rare diseases, but it is not only the investment we spent developing it, but also the drug. It also reflects the investment we have made to make it available and continue to support the community. Step. “The reason research is being done is because it is important to have data. They are safe and shinobi We are closely monitoring tolerability and that’s why this is really the way to go. Providing free medication for this clinical trial to help understand the impact on people using STXBP1 and SLC6A1. “We are a company with rare diseases. It is very important for us to support the community and that is why we support this trial,” he added. HIPAA, other patients’ experience in Horizon cannot be extended, but SLC6A1 Connect disagrees with their statement. The decision to access a life-saving drug seeks assurance that it will be made between the patient and his doctor. Horizon should not direct Ravicti-based access. SLC6A1 Connect is working to raise more than $ 4 million to fund research into gene replacement therapies that may treat SLC6A1. So far, we have raised about $ 2.5 million. Click to donate. k here.

A family in Kansas City, Missouri, hopes that existing medications can help his son’s rare illness. However, it costs more than $ 700,000 a year, so I’m worried that it may be out of reach.

“Charlie was a rough few months,” said Kim Fry.

Fry said her life was consumed by finding relief for her 3-year-old son suffering from a rare genetic disorder.

“95% of rare diseases, including Charlie, do not have an approved treatment,” she said.

This disorder is called SLC6A1 and is named after the affected gene. It causes seizures and delayed development.

“He’s taking epilepsy medication and we have a lot of really difficult side effects,” Fry said.

As she and other families raise money to fund gene replacement research, another drug is promising.

“SLC6A1Connect has funded a research study to test approved FDA drugs to see if they can hit anything that can help children. Ravicti returns as a really powerful option. I’ve done it, “says Fry. “We were very excited and gave the whole family great hope of getting a safe FDA-approved drug with few side effects.”

Ravicti is manufactured by Horizon Therapeutics and is donating the drug to an 8-week trial in children with seizure disorders such as SLC6A1. The drug price tag made Fly worried about what would happen at the end of the test.

“It’s $ 740,000 a year, more than $ 2,000 a day,” she said. “It’s not just an option.”

Charlie’s disability has not yet been approved and is not covered by insurance at this time.

“Knowing that there is something that might help Charlie is really devastating and emotional, and I don’t have access to it,” Fry said.

She said the company wants to work with families like her to make the drug more affordable.

“We want our children to be healthy and happy and have the same chances of life as everyone else,” she said.

Horizon Therapeutics said it was waiting for the results of the trial before deciding whether to expand access to the drug. In a statement to KMBC 9, Horizon said:

“”I would like to reiterate that this is a researcher-led trial and that we are offering the drug for free. We worked closely with the investigator to confirm that there are routes for investigators to request treatment beyond the trial period. It is based on the investigator’s clinical evaluation and oversight by the Institutional Review Board (IRB). Horizon does not deny access to our drug by anyone in this trial.

“Horizon is a company that is deeply involved with people with rare diseases. We have a long track record of doing the right thing and giving patients access to our medicines. That’s what we do in this situation. That’s what we’re doing at. “

Matt Flech, Vice President of Communications and Patient Support at Horizon, told KMBC9 on the phone: It is priced as a treatment for rare diseases due to the very small number of patients, but it reflects not only the investment spent on development, but also the investment spent on making the treatment available and supporting the community. I have. “

He said the trial would determine the next step.

“The reason the study is being done is because it’s important to have the data. They’re closely monitoring safety and tolerability, so this is really the way to go. We’re in this clinical trial. To provide the drug for free, to help understand its effects in people living with STXBP1 and SLC6A1, “Fresh said.

“We are a rare disease company. It is very important for us to support the community and that is why we support this trial,” he added.

In response, SLC6A1Connect sent a statement similar to the following:

“”Due to HIPAA, other patients’ experience in Horizon cannot be extended, but SLC6A1Connect disagrees with their statement. We seek assurance that the decision to use a life-saving drug will be made between the patient and his doctor. Horizon should not direct access to Ravicti based on any price set to protect the financial moat. “

SLC6A1 Connect continues to work to raise more than $ 4 million to fund research into gene replacement therapies that may treat SLC6A1. So far, we have raised about $ 2.5 million. To donate click here.

Kansas City family worries cost could keep drug from helping son with rare disorder Source link Kansas City family worries cost could keep drug from helping son with rare disorder

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