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

7 articles from the last 30 days matching "nerve cells"

ResearchRSS4 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 →
ResearchRSS4 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 →
ResearchRSS4 days ago

Early nerve-muscle connection flaws set stage for SMA damage

Scientists studied how spinal muscular atrophy (SMA) damages the connections between nerves and muscles. They found that in people with SMA, these nerve-muscle connections don't develop properly right after birth, making them weak and unable to work well before nerve cells start dying. This early problem may be an important part of how SMA causes muscle weakness.

WHY IT MATTERSUnderstanding that nerve-muscle connection problems happen early in SMA—before nerve cell loss—could lead to new treatments that target this early stage rather than only treating later damage.
Good to knowSpinal Muscular Atrophy (SMA)Read →
ResearchRSS4 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 →
ResearchRSS4 days ago

Antipsychotic drug may help treat SMA symptoms, research shows

Scientists found that haloperidol, a medication normally used to treat psychiatric conditions, may help treat spinal muscular atrophy (SMA) by increasing levels of a protein called SMN that is missing or low in SMA patients. In laboratory tests with mouse cells and human patient cells, haloperidol helped nerve cells survive longer, reduced harmful inflammation, and improved movement. This suggests haloperidol could potentially be used alongside or instead of current SMA treatments.

WHY IT MATTERSIf haloperidol proves effective in human trials, SMA patients could potentially benefit from a medication that already exists and is FDA-approved, potentially offering a faster path to treatment than developing entirely new drugs.
💬 Ask your doctorspinal muscular atrophyRead →
ResearchRSS4 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 →
ResearchPUBMEDApr 10

International neuroblastoma risk group consortium: a model of networking for rare cancers.

Doctors from around the world created a network called the International Neuroblastoma Risk Group to share information about neuroblastoma, a type of cancer that starts in nerve cells. They combined data from over 25,000 patients to help researchers understand the disease better and develop better treatment plans. This teamwork model shows how rare cancer research can be improved when hospitals and countries work together.

WHY IT MATTERSPatients with neuroblastoma now benefit from standardized treatment approaches and risk classifications developed through this international collaboration, which means more consistent and potentially better care regardless of where they receive treatment.
Good to knowneuroblastomaRead →

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