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rare disease

A collection of 11 posts

Twelve Extra Hours

Twelve Extra Hours

Disc Medicine says bitopertin met both goals in a phase 3 trial for a rare sunlight disease: 41.4 percent less protoporphyrin, and 12.2 more hours a month in light without pain. The FDA refused the drug once already.

An Oral Drug Eased Hereditary Angioedema Attacks in 1.28 Hours

hereditary angioedema

An Oral Drug Eased Hereditary Angioedema Attacks in 1.28 Hours

A Lancet phase 3 trial found an oral drug eased hereditary angioedema attacks in a median 1.28 hours, compared with more than 12 hours on placebo.

Gene Therapy Shows Promise for Limb-Girdle Muscular Dystrophy

Health & Medicine

Gene Therapy Shows Promise for Limb-Girdle Muscular Dystrophy

Atamyo's ATA-200 shows clinical benefits with no serious side effects in early trial

FDA Proposes New Framework to Speed Rare Disease Gene Therapy Approvals

Health & Medicine

FDA Proposes New Framework to Speed Rare Disease Gene Therapy Approvals

"Baby KJ" case demonstrates how personalized CRISPR therapy could become accessible to millions with rare diseases

New Seizure Drug Cuts Epilepsy Attacks by 91% in Children

epilepsy treatment

New Seizure Drug Cuts Epilepsy Attacks by 91% in Children

- New drug reduces seizures by 91% in children with rare, treatment-resistant epilepsy, offering breakthrough hope for families with limited treatment options.

Thyroid Eye Disease Treatment Shows Major Clinical Success

medical breakthrough

Thyroid Eye Disease Treatment Shows Major Clinical Success

Phase 3 trial shows elegrobart significantly improves thyroid eye disease symptoms, offering new hope for patients with this disfiguring condition.

Sildenafil Shows Promise for Devastating Childhood Disease

Sildenafil Shows Promise for Devastating Childhood Disease

Sildenafil Shows Promise for Devastating Childhood Disease Common erectile dysfunction drug offers hope for rare genetic condition A drug familiar to millions for treating erectile dysfunction has shown remarkable promise against one of childhood's most devastating diseases. Researchers at Charité-Universitätsmedizin Berlin found that sildenafil—the active ingredient in

FDA Approves Voyxact for Kidney Disease After Positive Trial

Health & Medicine

FDA Approves Voyxact for Kidney Disease After Positive Trial

First new treatment option offers hope for millions with IgA nephropathy

New Drug Cuts Seizures by 91% in Children with Rare Epilepsy

Health & Medicine

New Drug Cuts Seizures by 91% in Children with Rare Epilepsy

A groundbreaking new treatment has achieved extraordinary results in treating rare childhood epilepsy, reducing seizures by up to 91% in clinical trials. For families dealing with treatment-resistant epilepsy—where children can experience hundreds of seizures daily—this represents a potentially life-changing medical advance. The dramatic seizure reduction offers hope for

European Commission Approves Breakthrough Treatment for Rare Muscle Disease — March 7, 2026

New therapy offers hope for myasthenia gravis patients across Europe

European Commission Approves Breakthrough Treatment for Rare Muscle Disease

European Commission Approves Breakthrough Treatment for Rare Muscle Disease

European Commission approves new add-on therapy for myasthenia gravis, offering hope for patients with the rare autoimmune neuromuscular disorder.