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Genetic Disorders

Spinal Muscular Atrophy Rare Diseases Gout Heart Conditions FLVCR1 Variants SOD1 Mutations Antisynthetase Syndrome Congenital Deafness Thrombocytopenia Marfan Syndrome

5 ARTICLES

2mo ago

Oxford and Moderna Set to Launch Phase‑1 Trial of Preventive mRNA Vaccine for Lynch Syndrome

Researchers will test whether the vaccine mRNA‑4194 can safely provoke immune responses that stop pre‑cancerous cells in people with Lynch syndrome from becoming tumours.

4 ARTICLES

2mo ago

Brazil Expands Newborn Heel‑Prick Program but Access Remains Uneven

6 ARTICLES

2mo ago

Barcelona Team Performs First In‑Utero Repair of Severe Gastroschisis in Europe

3 ARTICLES

5mo ago

Brazil Treats First Infant in In‑Country Gene Therapy Trial for Severe Spinal Muscular Atrophy

10 ARTICLES

5mo ago

FDA Proposes ‘Plausible Mechanism’ Pathway for Individualized Rare-Disease Therapies

7 ARTICLES

5mo ago

Anti-Seizure Drug Levetiracetam Blocks Early Alzheimer’s Protein Production, Study Finds

3 ARTICLES

6mo ago

Rapid Zebrafish Test Classifies Newborn SMN1 Variants in Days

6 ARTICLES

8mo ago

FDA Approves Novartis’ Itvisma for SMA Patients Ages 2 and Up

4 ARTICLES

8mo ago

Study Reveals How Dialysis-Linked Kidney Cancers Develop, Marking a Distinct Disease Pathway

5 ARTICLES

last yr.

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

10 ARTICLES

last yr.

Eli Lilly’s Lepodisiran Achieves 94% Reduction in Genetic Heart Disease Risk Factor in Phase 2 Trial

8 ARTICLES

last yr.

Study Suggests Long-Term Anti-Amyloid Therapy May Delay Early-Onset Alzheimer’s

6 ARTICLES

last yr.

Gene Therapy Restores Sight in Children Born Blind Due to Rare Eye Disorder

3 ARTICLES

last yr.

IVF Linked to Slightly Higher Risk of Major Heart Defects in Babies

Genetic Disorders

Oxford and Moderna Set to Launch Phase‑1 Trial of Preventive mRNA Vaccine for Lynch Syndrome

Brazil Expands Newborn Heel‑Prick Program but Access Remains Uneven

Barcelona Team Performs First In‑Utero Repair of Severe Gastroschisis in Europe

Brazil Treats First Infant in In‑Country Gene Therapy Trial for Severe Spinal Muscular Atrophy

FDA Proposes ‘Plausible Mechanism’ Pathway for Individualized Rare-Disease Therapies

Anti-Seizure Drug Levetiracetam Blocks Early Alzheimer’s Protein Production, Study Finds

Rapid Zebrafish Test Classifies Newborn SMN1 Variants in Days

FDA Approves Novartis’ Itvisma for SMA Patients Ages 2 and Up

Study Reveals How Dialysis-Linked Kidney Cancers Develop, Marking a Distinct Disease Pathway

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

Never miss stories about

Genetic Disorders

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

Prenatal Trisomy Screening Rates Surge Across German States

Madrid Detects First SCID Case Through Neonatal Screening Program

Eli Lilly’s Lepodisiran Achieves 94% Reduction in Genetic Heart Disease Risk Factor in Phase 2 Trial

Study Suggests Long-Term Anti-Amyloid Therapy May Delay Early-Onset Alzheimer’s

Gene Therapy Restores Sight in Children Born Blind Due to Rare Eye Disorder

IVF Linked to Slightly Higher Risk of Major Heart Defects in Babies

Never miss stories about

Genetic Disorders