NewsUNITERAREApr 3
The PAN Foundation's financial assistance fund for ALS (a disease that affects nerve cells controlling muscles) is currently closed and not accepting new applications. This fund previously provided up to $12,000 per year to help patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have ALS and were counting on this $12,000 annual assistance from PAN Foundation, you'll need to explore other financial resources immediately, as this fund is no longer available.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for people with primary immunodeficiency is currently closed and not accepting new applications. Primary immunodeficiency is a group of disorders where the immune system doesn't work properly, making it harder to fight infections. When this fund was open, it could help eligible patients pay up to $12,000 per year toward treatment costs.
WHY IT MATTERSPatients with primary immunodeficiency who were relying on this $12,000 annual assistance need to immediately identify alternative funding sources, as this program is no longer accepting applications as of April 2, 2026.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for Duchenne muscular dystrophy patients has closed as of April 2, 2026. This program previously provided up to $12,000 per year to help eligible patients pay for treatment costs. Patients who need financial help with their DMD care should contact PAN Foundation or explore other assistance programs.
WHY IT MATTERSPatients with Duchenne muscular dystrophy who were relying on this $12,000 annual benefit to afford medications or treatments now need to find alternative financial assistance sources immediately.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for idiopathic pulmonary fibrosis (a lung disease where lung tissue becomes scarred and stiff over time) is currently closed and is not accepting new applications. This program previously offered up to $12,000 per year to help patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have idiopathic pulmonary fibrosis and need help paying for medications or treatment, you cannot currently apply to this specific PAN Foundation program, so you'll need to explore other financial assistance options.
NewsUNITERAREApr 3
The PAN Foundation's Multiple Sclerosis Fund, which helped patients pay for MS medications and treatments, is currently closed and not accepting new applications. The fund previously offered up to $12,000 per year in financial assistance. The status changed on April 2, 2026, so it may reopen in the future.
WHY IT MATTERSIf you have MS and were relying on this fund to help pay for your medications, you'll need to find alternative financial assistance programs immediately since this specific fund is no longer accepting applications.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance fund for paroxysmal nocturnal hemoglobinuria (PNH) — a rare blood disorder where red blood cells break down too quickly — is currently closed and stopped accepting new applications as of April 2, 2026. This fund previously provided up to $12,000 per year to help patients pay for treatment costs. Patients who need financial help with their PNH medications should check the PAN Foundation website for updates on when the fund might reopen.
WHY IT MATTERSPatients with PNH who were relying on this $12,000 annual benefit to afford their medications now need to find alternative financial assistance programs immediately, as this major funding source is no longer available.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for spinal muscular atrophy (SMA) is currently closed and is not accepting new applications. This program previously provided up to $12,000 per year to help eligible patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have spinal muscular atrophy and need help paying for medications like Zolgensma or Spinraza, you'll need to explore other financial assistance programs since this major funding source is temporarily unavailable.
Clinical trialUNITERAREApr 3
Researchers are looking for people with diabetes to test a new way to treat bad breath caused by gum disease. The treatment combines regular gum cleaning with a special light therapy that kills bacteria in the mouth. This study will help doctors understand if this combination works better than standard gum treatment alone.
WHY IT MATTERSIf you have diabetes and struggle with bad breath despite good oral hygiene, this trial offers access to an innovative combination treatment that may address both the gum disease and halitosis that often accompany diabetes.
Clinical trialUNITERAREApr 3
Researchers at King's College London are testing whether probiotics (helpful bacteria) can reduce skin inflammation that some people experience during perimenopause (the years leading up to menopause). This is an early-stage study to see if this approach might work before larger trials are done. The study is now accepting participants.
WHY IT MATTERSIf probiotics prove effective for perimenopause-related skin inflammation, this could offer a non-hormonal treatment option for people experiencing this symptom during the menopausal transition.
Clinical trialUNITERAREApr 3
Researchers are looking for patients who need a procedure called a transjugular intrahepatic portosystemic shunt (TIPS) to help study how blood clotting works in patients with liver disease. This trial will examine blood clotting patterns before and after the procedure to better understand how TIPS affects the body's ability to form and break down blood clots. The study is recruiting patients starting in April 2026 and is being run by hospitals in Paris.
WHY IT MATTERSThis trial is now recruiting patients undergoing TIPS placement — if you have advanced liver disease with portal hypertension and are scheduled for this procedure, you may be eligible to contribute to research that could improve how doctors manage bleeding risks in liver disease patients.
Clinical trialUNITERAREApr 3
Researchers at New York State Institute for Basic Research are testing a new treatment called transcranial direct current stimulation (tDCS) in children with autism. This treatment uses mild electrical currents applied to the scalp to potentially help with autism symptoms. The study is now accepting children to participate and will begin in April 2026.
WHY IT MATTERSThis trial offers families with autistic children a chance to explore a non-medication brain stimulation approach that may help with core autism symptoms, with enrollment actively opening at a major research institution.
Clinical trialUNITERAREApr 3
Researchers are testing a new medicine called glycerol tributyrate to see if it can help people with two rare mitochondrial diseases: MELAS (a condition that causes brain inflammation, acid buildup in the blood, and stroke-like episodes) and LHON-Plus (a condition that damages the optic nerve and causes vision loss). This is an early-stage study that will enroll patients starting in April 2026.
WHY IT MATTERSThis is the first clinical trial testing glycerol tributyrate specifically in MELAS and LHON-Plus patients, offering a potential new treatment option for these currently untreatable mitochondrial disorders.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance fund for Gaucher disease patients is currently closed and is not accepting new applications. This fund previously provided up to $12,000 per year to help patients pay for treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have Gaucher disease and were counting on this $12,000 annual assistance from PAN Foundation, you'll need to explore other financial support options immediately, as this fund is no longer available.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for sickle cell disease patients is currently closed and is not accepting new applications. This program previously offered up to $12,000 per year to help eligible patients with treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have sickle cell disease and were counting on this $12,000 annual assistance from PAN Foundation, you'll need to explore other financial support options immediately since this fund is no longer accepting applications.
NewsUNITERAREApr 3
The PAN Foundation's financial assistance program for cystic fibrosis patients is currently closed and not accepting new applications. This program previously provided up to $12,000 per year to help eligible patients pay for their medications and treatment costs. The fund status changed on April 2, 2026.
WHY IT MATTERSIf you have cystic fibrosis and were counting on this $12,000 annual assistance from PAN Foundation, you'll need to explore alternative financial aid programs immediately to help cover your medication and treatment expenses.
🔴 BreakingDrug approvalRSSApr 3
The FDA has approved a new drug called Avlayah to treat Hunter syndrome, a rare genetic disease that affects the brain and nervous system. Hunter syndrome happens when the body can't break down certain substances properly, causing serious health problems over time. This approval means patients with Hunter syndrome now have a new treatment option available to help manage the neurologic symptoms of their condition.
WHY IT MATTERSThis is the first FDA approval of Avlayah specifically for the neurologic manifestations of Hunter syndrome, offering patients a new therapeutic option for managing brain and nervous system symptoms that were previously difficult to treat.
PolicyRSSApr 3
The FDA met with several states to discuss a program that lets states and Native American tribes buy prescription drugs from Canada at lower prices and bring them into the United States. This program, called section 804 importation, is designed to help Americans afford their medications. The meeting focused on how this program works and how states can participate.
WHY IT MATTERSPatients with rare diseases who take expensive prescription medications may be able to access more affordable versions of their drugs through this importation program, potentially reducing their out-of-pocket costs significantly.
PolicyRSSApr 3
The FDA created a new online tool called the FDA Adverse Event Monitoring System (AEMS) that makes it easier for people to look up safety reports about medicines and medical devices. This tool brings together information from different FDA databases into one place so patients and doctors can see what side effects or problems have been reported. The FDA says this is an important step toward being more transparent and honest about drug and device safety.
WHY IT MATTERSPatients with rare diseases can now directly search for safety reports on their specific medications and medical devices without waiting for their doctor to look it up, helping them make more informed decisions about their treatment.
🔴 BreakingDrug approvalRSSApr 3
The FDA approved a new drug called Foundayo (orforglipron) on a special fast-track program called the National Priority Voucher program. This is the fifth drug approved through this program, which helps speed up approval for medicines that address important health needs. The program is designed to get helpful new treatments to patients faster.
WHY IT MATTERSThis approval demonstrates the National Priority Voucher program is working to bring new treatment options to patients with rare diseases more quickly than the standard FDA review process.
PolicyRSSApr 3
The FDA is holding a public meeting to get feedback from patients, doctors, and companies about a pilot program called the Commissioner's National Priority Voucher. This program is designed to speed up the development of drugs for diseases that are considered national health priorities. The FDA wants to hear what people think about how the program is working and how it could be improved.
WHY IT MATTERSIf you have a rare disease, this meeting could influence which diseases the FDA prioritizes for faster drug development in the future—your input or your disease community's input could help shape which conditions get expedited attention.