Thank you to all the patients, families, caregivers, and the Immune Deficiency Foundation community for visiting us at the 2024 Primary Immunodeficiency (PI) Reel Talk conference and sharing your stories. We heard loud and clear about the urgent need for early, accurate diagnosis and targeted treatments. Your stories inspire and motivate us to continue our work at X4 Pharmaceuticals in bringing innovative treatments to the PI community. We look forward to seeing you again at upcoming walks and next year’s conference. #2024PIConference
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More Relevant Posts
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Have you checked out our medical information website? Explore our resources in oncology, including a convenient live chat function for your medical information questions during business hours. #oncology #medicalinformation #livechat #Eisai
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Check out our series of blog posts discussing USP 800 and its impact on healthcare organizations (cancer centers, pharmacies, hospitals). USP 800 hazardous drug compounding and safe handling standards become enforceable on November 1, 2023. https://bit.ly/3rbwJZL
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There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities
Avalere Health | Putting the patient first: Moving from patient communities to patient co-creation in rare disease
avalerehealth.com
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There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities
Avalere Health | Putting the patient first: Moving from patient communities to patient co-creation in rare disease
avalerehealth.com
-
There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities
Avalere Health | Putting the patient first: Moving from patient communities to patient co-creation in rare disease
avalerehealth.com
-
There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities
Avalere Health | Putting the patient first: Moving from patient communities to patient co-creation in rare disease
avalerehealth.com
-
There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities
Avalere Health | Putting the patient first: Moving from patient communities to patient co-creation in rare disease
avalerehealth.com
-
There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities
Avalere Health | Putting the patient first: Moving from patient communities to patient co-creation in rare disease
avalerehealth.com
-
There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities
Avalere Health | Putting the patient first: Moving from patient communities to patient co-creation in rare disease
avalerehealth.com
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What is Wainua? Wainua (eplontersen) is used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN), which is a rare, progressive disease that can be fatal if not treated. Amyloidosis is a disease caused by a buildup of abnormal proteins called amyloid. Wainua is from a class of medications called LICA (ligand-conjugated antisense oligonucleotide). Wainua works by decreasing the amount of TTR protein that is made, which slows disease progression and improves neuropathy and the patient's quality of life. Wainua is given as a monthly self-administered subcutaneous injection, using an autoinjector. Wainua received FDA approval on December 21, 2023, after positive results from the phase III clinical trial NEURO-TTRansform (NCT04136184).
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