Latest Advances

  • Condition: Spinal Muscle Atrophy (SMA)
  • Journal: Orphanet journal of rare diseases
  • Treatment Used: Oral Medications and One-Time Infusions
  • Number of Patients: 101
  • Published —
This study examined patient preference for spinal muscular atrophy (SMA) treatment.
  • Condition: Spinal Muscular Atrophy in Children
  • Journal: Scientific reports
  • Treatment Used: Magnetically Controlled Growing Rods
  • Number of Patients: 17
  • Published —
This study assessed the long term effects and spinal deformity control in spinal muscular atrophy (SMA) patients treated with magnetically controlled growing rod (MCGR) implants for up to four years.
  • Condition: Chronic Kidney Disease
  • Journal: The New England journal of medicine
  • Treatment Used: Finerenone
  • Number of Patients: 2833
  • Published —
In this study, researchers evaluated the outcomes of treating chronic kidney disease with finerenone.
  • Condition: Skeletal Muscle Cramps
  • Journal: The Cochrane database of systematic reviews
  • Treatment Used: Magnesium
  • Number of Patients: 735
  • Published —
This study investigated the use of magnesium to treat patients with skeletal muscle cramps.
  • Condition: Non-Invasive Ventilation (NIV) in Patients with Amyotrophic Lateral Sclerosis (ALS)
  • Journal: Scientific reports
  • Treatment Used: Gastrostomy Placement
  • Number of Patients: 92
  • Published —
This study determined the prognostic factors for survival after gastrostomy placement in routine non-invasive ventilation (NIV) users with amyotrophic lateral sclerosis (ALS), taking into consideration ventilator dependence.
  • Condition: Spinal Muscular Atrophy Type 3
  • Journal: The neurologist
  • Treatment Used: Nusinersen
  • Number of Patients: 1
  • Published —
This case report describes a patient with spinal muscular atrophy type 3 that was treated using nusinersen.
  • Condition: Amyotrophic Lateral Sclerosis
  • Journal: The New England journal of medicine
  • Treatment Used: Sodium Phenylbutyrate-Taurursodiol
  • Number of Patients: 137
  • Published —
In this study, researchers evaluated the safety and effectiveness of using sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis.
  • Condition: Long-term Neurological Conditions
  • Journal: JAMA network open
  • Treatment Used: Short-term Integrated Palliative Care
  • Number of Patients: 350
  • Published —
In this study, researchers evaluated the outcomes of short-term integrated palliative care for patients with long-term neurological conditions.
  • Condition: Chronic Respiratory Failure
  • Journal: BMC pulmonary medicine
  • Treatment Used: Home Mechanical Ventilation
  • Number of Patients: 74
  • Published —
This study tested the safety and efficacy of a home-based mechanical ventilation to treat patients with chronic respiratory failure.
  • Condition: Amyotrophic Lateral Sclerosis
  • Journal: International journal of medical sciences
  • Treatment Used: Autologous Lineage-negative Stem/progenitor Cell Therapy
  • Number of Patients: 12
  • Published —
In this study, researchers evaluated the outcomes of using autologous lineage-negative stem/progenitor cell therapy for the treatment of speech and swallowing deficits from amyotrophic lateral sclerosis.
  • Condition: Risk of Amyotrophic Lateral Sclerosis (ALS)
  • Journal: Scientific reports
  • Treatment Used: Proton Pump Inhibitors (PPIs)
  • Number of Patients: 2484
  • Published —
This study assessed the link between proton pump inhibitors (PPIs) use with the risk of developing amyotrophic lateral sclerosis (ALS).
  • Condition: Amyotrophic Lateral Sclerosis (ALS)
  • Journal: Cells
  • Treatment Used: Repeated Application of Autologous Bone Marrow-Derived Lineage-Negative Stem/Progenitor Cells
  • Number of Patients: 40
  • Published —
This study evaluated three intrathecal administrations of autologous bone marrow-derived lineage-negative (Lin-) cells every six weeks in patients with sporadic amyotrophic lateral sclerosis (ALS).
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