Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

8 articles from the last 7 days matching "als"

ResearchRSSYesterday

Keeping weight steady after tube feeding may help ALS survival

A study of 121 people with ALS found that those who kept their weight more stable after starting tube feeding lived longer. This was true even if they weren't eating a lot of calories when they first started the tube feeding. The finding suggests that preventing weight loss after tube feeding may help people with ALS live longer.

WHY IT MATTERSIf you or a loved one with ALS is considering or has started tube feeding, this research suggests that working with your care team to maintain steady weight—not just calorie intake—could potentially extend survival.
💬 Ask your doctorAmyotrophic Lateral SclerosisRead →
ResearchRSS3 days ago

High pulmonary resistance indicates worse outcomes in new PH study

A study from Japan found that people with pulmonary hypertension (a condition where blood vessels in the lungs become stiff and narrow) who have higher pulmonary vascular resistance—a measure of how hard the heart has to work to pump blood through the lungs—tend to have worse health outcomes. The study also looked at whether a type of diabetes medication called SGLT2 inhibitors might help these patients.

WHY IT MATTERSIf you have pulmonary hypertension related to heart disease, knowing that high pulmonary vascular resistance predicts worse outcomes could help your doctor decide whether to start more aggressive treatment earlier or monitor you more closely.
💬 Ask your doctorpulmonary hypertension due to left heart diseaseRead →
ResearchRSS3 days ago

Study links protein, fat regulator to nerve cell damage in ALS

Scientists discovered that a protein called UBQLN2 helps nerve cells manage both proteins and fats. When UBQLN2 doesn't work properly — especially in controlling fats — it may cause nerve cells to die in ALS, a disease that weakens muscles over time. This discovery suggests doctors might be able to help nerve cells stay healthier by fixing how fats are controlled.

WHY IT MATTERSUnderstanding how fat regulation breaks down in ALS nerve cells could lead to new treatments that target this specific problem, potentially slowing or stopping nerve cell damage in people living with ALS.
Good to knowamyotrophic lateral sclerosisRead →
ResearchRSS3 days ago

Exposure to pesticide HCB may raise risk of ALS, new study finds

A new study from Denmark found that people with higher levels of HCB (a pesticide that was banned in many countries) in their blood may have a higher risk of developing ALS, a disease that affects nerve cells and causes muscle weakness. HCB was used as a pesticide in the past but is no longer allowed in many places because it can be harmful to health. This research suggests that exposure to this old pollutant might be connected to ALS risk.

WHY IT MATTERSIf you have ALS or a family history of it, understanding environmental risk factors like HCB exposure could help you and your doctor identify potential causes and discuss ways to reduce exposure to similar pollutants.
💬 Ask your doctoramyotrophic lateral sclerosisRead →
ResearchRSS3 days ago

Simple nerve signals may help track disease progression in SMA

Researchers found that a simple electrical test using surface electrodes on the skin can measure nerve signals in people with spinal muscular atrophy (SMA). These signals were much weaker in SMA patients compared to healthy people, which suggests this test could be a useful way to track how the disease changes over time.

WHY IT MATTERSIf validated, this non-invasive surface electrode test could provide a faster, easier, and cheaper way to monitor SMA progression in clinical trials and patient care compared to current muscle strength assessments.
Good to knowspinal muscular atrophyRead →
ResearchRSS3 days ago

Long-term air pollution exposure shows no clear link to ALS in UK study

A large study in the UK followed over 500,000 people for about 10 years and found that breathing polluted air for a long time does not appear to increase the risk of getting ALS, a disease that affects nerve cells that control muscles. Fewer than 1,000 people in the study developed ALS during the study period. This suggests that air pollution may not be an important risk factor for developing ALS.

WHY IT MATTERSIf you have ALS or are worried about developing it, this study suggests that long-term air pollution exposure is unlikely to be a major cause, which may help reduce anxiety about environmental risk factors you cannot easily control.
Good to knowamyotrophic lateral sclerosisRead →
ResearchRSS3 days ago

Spontaneous mutations may underlie some sporadic ALS cases

Scientists discovered that some people with ALS (a disease that affects nerve cells controlling muscles) may develop the condition from new mutations that happen by chance in their nerve cells, rather than inheriting the mutation from their parents. This is different from inherited ALS cases where a parent passes down a faulty gene. This finding suggests there are multiple ways ALS can develop, which could help doctors better understand and treat the disease.

WHY IT MATTERSIf your ALS diagnosis is sporadic (not inherited), this research suggests your condition may have developed from random mutations in your nerve cells rather than a genetic predisposition, which could change how doctors approach your treatment and genetic counseling.
Good to knowAmyotrophic Lateral Sclerosis (ALS)Read →
ResearchBIORXIV5 days ago

Preprint: Blood-based RNA-Seq of 5412 individuals with rare disease identifies new candidate diagnoses in the National Genomic Research Library

Researchers analyzed blood samples from over 5,400 people with rare genetic diseases to see if a test called RNA-Seq could help find the genetic cause of their conditions. They found that this blood test works better for some diseases than others, and they used special computer programs to spot unusual gene activity patterns that might explain why people got sick. This study shows that blood tests could be a useful tool to help diagnose rare diseases alongside other genetic tests.

WHY IT MATTERSIf you have an undiagnosed rare disease, this research suggests blood-based RNA testing could help identify the genetic cause—potentially leading to a diagnosis after years of searching.
Good to knowRead →

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases