Nervous System Diseases

The Service de Neurologie at Raymond‑Poincare Hospital conducts research on conditions such as spinal muscular atrophy, generalized myasthenia gravis, lower‑limb spasticity and neurogenic bladder, aiming to test novel therapies and improve functional outcomes for patients.

  • Spinal muscular atrophy disease‑modifying treatments
  • Myasthenia gravis antibody‑targeted interventions
  • Botulinum toxin for lower‑limb spasticity
  • Neurogenic bladder management in multiple sclerosis and spinal cord injury
  • Safety assessments of emerging neurological agents

These studies combine clinical expertise with patient‑centered design to generate reliable data on efficacy and long‑term safety, supporting future therapeutic options.

Metabolic & Genetic Disorders

Research efforts target rare metabolic conditions, notably Fabry disease and late‑onset Pompe disease, evaluating enzyme replacement enhancements, pain relief strategies, and cardiac impact of new molecules.

  • Fabry disease pain and organ involvement studies
  • Long‑term safety of adjunct therapies for Pompe disease
  • Venglustat effects on left ventricular mass in Fabry patients
  • Combination approaches with enzyme replacement therapy
  • Quality‑of‑life assessments in hereditary metabolic disorders

The goal is to broaden therapeutic options and provide evidence for improved disease management in these underserved patient groups.

Infectious Diseases

The site explores both viral and bacterial infections, including HIV and HBV co‑infection, hospital‑acquired pneumonia, sepsis, and recurrent urinary tract infections, focusing on innovative antiviral combinations and antibiotic‑sparing strategies.

  • Broadly neutralizing antibodies combined with antiretroviral therapy for HIV
  • HBV control strategies in patients on triple therapy
  • Dual antiretroviral regimens for sustained viral suppression
  • Antibiotic stewardship in severe pneumonia
  • Prophylactic approaches to urinary tract infections in neurogenic bladder

These trials aim to reduce disease burden, limit drug resistance, and improve survival outcomes for vulnerable populations.

Cardiovascular Diseases

Within the metabolic disease programs, cardiovascular health is a key focus, especially the impact of therapies on cardiac remodeling in Fabry disease.

  • Assessment of left ventricular mass reduction with novel agents
  • Monitoring of cardiac biomarkers during metabolic disease treatment
  • Evaluation of long‑term cardiovascular safety in enzyme‑based therapies

By linking metabolic and cardiac endpoints, the research supports comprehensive care for patients at risk of heart complications.

Respiratory Tract Diseases

Studies address severe lung infections such as hospital‑acquired pneumonia and sepsis, testing anti‑inflammatory regimens and optimized antibiotic use.

  • Dexamethasone plus standard care for severe pneumonia
  • Evaluation of mortality and discharge time in critical respiratory illness
  • Safety monitoring of adjunctive therapies in sepsis

The objective is to improve survival rates and accelerate recovery for patients with acute respiratory conditions.

Immune System Diseases

Clinical investigations include autoimmune disorders like generalized myasthenia gravis, where novel antibodies and immunomodulators are being assessed for symptom control.

  • ALXN1720 efficacy in myasthenia gravis
  • DNTH103 safety and tolerability in generalized myasthenia gravis
  • Autoantibody profiling to guide personalized therapy

These projects aim to refine treatment algorithms and reduce disease‑related disability.

Pathological Conditions, Signs & Symptoms

Pain, spasticity, and other functional symptoms are evaluated across several trials, with an emphasis on patient‑reported outcomes and functional improvement.

  • Neuropathic and abdominal pain relief in Fabry disease
  • Botulinum toxin efficacy for lower‑limb spasticity
  • Quality‑of‑life measures in metabolic and neurological disorders

The research seeks to translate symptom reduction into meaningful daily‑life benefits for participants.

Bacterial Infections & Mycoses

Targeted investigations address recurrent bacterial urinary infections, sepsis, and peri‑operative infection control, exploring both prophylactic and therapeutic options.

  • OM‑89 versus placebo for reducing antibiotic courses in neurogenic bladder
  • Peri‑operative antibiotic strategy for asymptomatic bacteriuria during botulinum toxin injections
  • Safety and dosing studies of novel agents in sepsis and coagulopathy

The aim is to limit infection recurrence, decrease antibiotic exposure, and enhance patient safety.

Virus Diseases

Viral research centers on HIV‑1 primary infection, chronic HIV‑HBV co‑infection, and strategies to achieve sustained viral remission without continuous therapy.

  • Dual long‑acting broadly neutralizing antibodies with ART
  • Maintenance dual antiretroviral therapy with cabotegravir/lenacapavir
  • HBV virological control after treatment reduction in HIV‑HBV co‑infected patients

These trials explore pathways to long‑term viral control, potentially reducing lifelong medication burden.