Adrenoleukodystrophy

Children's Hospital of Philadelphia and Sidra Medicine to Establish First-Ever Pediatric Bone Marrow Transplant Program in Qatar

Retrieved on: 
Martedì, Maggio 14, 2024
Patient, CHOP, Business, Disorder, Transplant, BMT, Immune system, Sickle cell disease, FDA, DSM-IV codes, Lymphoma, Immunodeficiency, Blood, Research, Cancer, Pediatrics, Adrenoleukodystrophy, Leukemia, Children's Hospital of Philadelphia, Hematology, Medicine, Hospital, Woman, B cell, Thalassemia, MOU, CTTS, Beta thalassemia

The MOU, signed by Dr. Iyabo Tinubu-Karch, CEO of Sidra Medicine, and Madeline Bell, President and CEO of Children's Hospital of Philadelphia will establish a collaboration between CHOP and Sidra Medicine in Sidra Medicine's efforts to create the first-ever pediatric bone marrow transplant (BMT) program in Qatar.

Key Points: 
  • The MOU, signed by Dr. Iyabo Tinubu-Karch, CEO of Sidra Medicine, and Madeline Bell, President and CEO of Children's Hospital of Philadelphia will establish a collaboration between CHOP and Sidra Medicine in Sidra Medicine's efforts to create the first-ever pediatric bone marrow transplant (BMT) program in Qatar.
  • The main objective of the MOU is to establish a working relationship between CHOP's Cellular Therapy and Transplant Section (CTTS) and Sidra Medicine.
  • CHOP's Blood and Marrow Transplant program is part of CTTS and serves the needs of children who require a blood or marrow transplant to replace stem cells to treat their illness.
  • Sidra Medicine is the only hospital in Qatar to provide pediatric hematology and oncology care for children and young people with cancer and blood disorders.

Poxel Provides a Financial Update for the First Quarter 2024 and Announces the Postponement of its 2023 Full-Year Results Release

Retrieved on: 
Lunedì, Aprile 29, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, Diabetes, Clinical Trials, Other Health, USD, Patient, Adenosine, DSM-IV codes, ADPKD, Sumitomo Pharma, Safety, Pharmacokinetics, European Patent Convention, JPY, Merck Serono, Kidney, Male, Adrenoleukodystrophy, VLCFA, EUR, Plasma, Serono, Disease, NASH, Fatty liver disease, Patent, ALD, AMN, POC, Biomarker, Merck, EPO, Pharmaceutical industry

As a result, TWYMEEG gross sales should exceed its FY 2023 guidance of JPY 4.2 billion (EUR 25.7 million)3 driven by a favourable sales trend in Japan.

Key Points: 
  • As a result, TWYMEEG gross sales should exceed its FY 2023 guidance of JPY 4.2 billion (EUR 25.7 million)3 driven by a favourable sales trend in Japan.
  • Beyond 2024, Poxel expects to receive escalating double-digit royalties as well as additional sales-based payments upon achievement of contractually based sales thresholds.
  • For territories not covered by its agreement with Sumitomo Pharma, Poxel is in ongoing discussions with potential partners for Imeglimin.
  • Net financial debt (excluding IFRS16 impacts and derivative debts) was EUR 47.4 million as of March 31, 2024, as compared to EUR 45.6 million as of December 31, 2023.

TeleRare Health™ Announces Launch of National Virtual Clinic for Rare Disease Patients

Retrieved on: 
Martedì, Aprile 30, 2024
Wilson's disease, Bangladesh Technical Education Board, Genomics, Kidney, SCID, Diagnosis, Safety, NIH, AI, Heart, Adrenoleukodystrophy, MD, Partnership, Children's National Hospital, DMD, FXS, Research, Human, Genetic testing, Disease, Patient, ALD, Pediatrics, Krabbe disease, Duchenne muscular dystrophy, PD, GMG, Fragile X syndrome, Pharmaceutical industry

MINNEAPOLIS, April 30, 2024 /PRNewswire/ -- 8:00a (CET) April 30, 2024 - TeleRare Health™ launched today, a virtual clinic for Rare Disease patients and their families.

Key Points: 
  • MINNEAPOLIS, April 30, 2024 /PRNewswire/ -- 8:00a (CET) April 30, 2024 - TeleRare Health™ launched today, a virtual clinic for Rare Disease patients and their families.
  • TeleRare Health aims to be the premier provider of virtual care for rare and genomic disease nationwide, envisioning a world where all patients have access to cutting edge diagnostics, treatments, and specialists.
  • "Significant breakthroughs have occurred in the management of rare disease," said Alex Katz, MD, medical geneticist and Chief Medical Officer of TeleRare Health.
  • We're overdue for a virtual clinic for the millions of Americans with rare disease conditions, and TeleRare Health's launch is cause for optimism.

Rare Disease Day 2024 - Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy - special film launched

Retrieved on: 
Giovedì, Febbraio 29, 2024
Rare disease, CALD, Central nervous system, Adrenoleukodystrophy, Committee, Leukodystrophy, Hospital, X-ALD, PPAR, Therapy, CHMP, CNS, European Medicines Agency, Patient, Disease, Pharmaceutical industry

The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.

Key Points: 
  • The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.
  • Dr. Jörg-Thomas Dierks, Chief Executive Officer of Neuraxpharm, said: "The impact this disease has on patients and their families cannot be overstated.
  • We hope that this film will raise awareness and understanding of the condition."
  • Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD.

Rare Disease Day 2024 - Call for greater awareness of X-linked adrenoleukodystrophy and cerebral adrenoleukodystrophy - special film launched

Retrieved on: 
Giovedì, Febbraio 29, 2024
Rare disease, CALD, Central nervous system, Adrenoleukodystrophy, Committee, Leukodystrophy, Hospital, X-ALD, PPAR, Therapy, CHMP, CNS, European Medicines Agency, Patient, Disease, Pharmaceutical industry

The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.

Key Points: 
  • The film can be found on Neuraxpharm's and Minoryx's websites, YouTube and LinkedIn channels.
  • Dr. Jörg-Thomas Dierks, Chief Executive Officer of Neuraxpharm, said: "The impact this disease has on patients and their families cannot be overstated.
  • We hope that this film will raise awareness and understanding of the condition."
  • Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD.

Poxel Reports Cash and Revenue for the Full Year 2023 and Provides Corporate Update

Retrieved on: 
Giovedì, Febbraio 15, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Merck, Kidney, USD, POC, Adrenoleukodystrophy, Merck Serono, EPO, JPY, AMN, Sumitomo Pharma, European Patent Convention, Safety, Biomarker, EUR, ALD, ADPKD, Serono, Adenosine, Pharmacokinetics, Patent, Fatty liver disease, Patient, Diabetes, Disease, NASH, Plasma, Male, DSM-IV codes, VLCFA, Pharmaceutical industry

Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We remain focused and continue to concentrate all our efforts at securing financing options, prioritizing non-dilutive solution, and partnerships.

Key Points: 
  • Thomas Kuhn, Chief Executive Officer of Poxel, stated: “We remain focused and continue to concentrate all our efforts at securing financing options, prioritizing non-dilutive solution, and partnerships.
  • We’ve also progressed our advanced discussions – in some cases on an exclusive basis – with several potential partners for each of our three proprietary products.
  • Based on this conservative assumption, Poxel expects to receive 8% royalties on TWYMEEG® net sales.
  • As of December 31, 2023, total cash and cash equivalents were EUR 2.3 million (USD 2.6 million)1, as compared to EUR 13.1 million (USD 14.0 million) as of December 31, 2022.

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 January 2024

Retrieved on: 
Domenica, Febbraio 4, 2024
Civil service commission, Pneumonia, Febrile neutropenia, Eli Lilly, Triprolidine, International, Bacteria, Schizophrenia, Myelopathy, Incidence, ALD, Cefepime, Allergic rhinitis, Nasal congestion, Neutrophil, Agency, COMP, Fever, HAP, EEIG, Headache, Static pressure, Medicine, Committee, Conditional, Spinal cord, Hospital-acquired pneumonia, PTC, Neutropenia, Public, CHMP, European, AMD, Patient, Diagnosis, Marketing, RCVS, European Commission, Brain, Disease, Infection, Inflammation, Male, PRES, PRAC, Pain, Pyelonephritis, Urinary tract infection, VAP, Ageing, EMA, Geographic atrophy, Adrenoleukodystrophy, Therapy, News, INN, Duchenne muscular dystrophy, Common, Fungal infection, Vaccine

Three new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended three medicines for approval at its January 2024 meeting.The committee recommended…, Positive recommendations on new medicines, Exblifep International non-proprietary name (INN)cefepime / enmetazobactam Marketing-authorisation applicantAdvanz Pharma LimitedTherapeutic indicationTreatment of: 1)…, Ryzneuta INNefbemalenograstim alfaMarketing-authorisation applicantEvive Biotechnology Ireland LimitedTherapeutic indicationReduction in the duration of neutropenia and…, Positive recommendations on new generic medicines, Niapelf INNpaliperidoneMarketing-authorisation applicantNeuraxpharm Pharmaceuticals S.L.Therapeutic indicationTreatment of schizophreniaMore informationNiapelf: Pending…, Negative recommendations on new medicines, Nezglyal INNleriglitazoneMarketing-authorisation applicantMinoryx Therapeutics S.L.Therapeutic indicationTreatment of cerebral progression and myelopathy in male…, Syfovre INNpegcetacoplanMarketing-authorisation applicantApellis Netherlands B.V.Therapeutic indicationTreatment of geographic atrophy (GA) secondary to age-related…, Positive recommendations on extensions of indications, Abecma INNidecabtagene vicleucel Marketing-authorisation holderBristol-Myers Squibb Pharma EEIGMore informationAbecma: Pending EC decision, Prevenar 20 (previously Apexxnar) Common namepneumococcal polysaccharide conjugate vaccine (20-valent, adsorbed)Marketing-authorisation holderPfizer Europe MA EEIGMore…, Aspaveli INNpegcetacoplanMarketing-authorisation holderSwedish Orphan Biovitrum AB (publ)More informationAspaveli: Pending EC decision, Retsevmo INNselpercatinibMarketing-authorisation holderEli Lilly Nederland B.V.More informationRetsevmo: Pending EC decision, Outcome of re-examination of recommendation on non-renewal of Conditional Marketing Authorisation, Translarna INNataluren Marketing-authorisation holderPTC Therapeutics International LimitedNewsEMA confirms recommendation for non-renewal of authorisation of Duchenne…, Outcome of referral, Pseudoephedrine Marketing-authorisation holderVarious companiesMore informationPseudoephedrine-containing medicinal products, Re-examination of conclusion of referral, Synapse Labs Pvt.

Key Points: 
  • Three new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended three medicines for approval at its January 2024 meeting.The committee recommended…, Positive recommendations on new medicines, Exblifep International non-proprietary name (INN)cefepime / enmetazobactam Marketing-authorisation applicantAdvanz Pharma LimitedTherapeutic indicationTreatment of: 1)…, Ryzneuta INNefbemalenograstim alfaMarketing-authorisation applicantEvive Biotechnology Ireland LimitedTherapeutic indicationReduction in the duration of neutropenia and…, Positive recommendations on new generic medicines, Niapelf INNpaliperidoneMarketing-authorisation applicantNeuraxpharm Pharmaceuticals S.L.Therapeutic indicationTreatment of schizophreniaMore informationNiapelf: Pending…, Negative recommendations on new medicines, Nezglyal INNleriglitazoneMarketing-authorisation applicantMinoryx Therapeutics S.L.Therapeutic indicationTreatment of cerebral progression and myelopathy in male…, Syfovre INNpegcetacoplanMarketing-authorisation applicantApellis Netherlands B.V.Therapeutic indicationTreatment of geographic atrophy (GA) secondary to age-related…, Positive recommendations on extensions of indications, Abecma INNidecabtagene vicleucel Marketing-authorisation holderBristol-Myers Squibb Pharma EEIGMore informationAbecma: Pending EC decision, Prevenar 20 (previously Apexxnar) Common namepneumococcal polysaccharide conjugate vaccine (20-valent, adsorbed)Marketing-authorisation holderPfizer Europe MA EEIGMore…, Aspaveli INNpegcetacoplanMarketing-authorisation holderSwedish Orphan Biovitrum AB (publ)More informationAspaveli: Pending EC decision, Retsevmo INNselpercatinibMarketing-authorisation holderEli Lilly Nederland B.V.More informationRetsevmo: Pending EC decision, Outcome of re-examination of recommendation on non-renewal of Conditional Marketing Authorisation, Translarna INNataluren Marketing-authorisation holderPTC Therapeutics International LimitedNewsEMA confirms recommendation for non-renewal of authorisation of Duchenne…, Outcome of referral, Pseudoephedrine Marketing-authorisation holderVarious companiesMore informationPseudoephedrine-containing medicinal products, Re-examination of conclusion of referral, Synapse Labs Pvt.
  • Ltd. Marketing-authorisation holderVarious companiesMore informationSynapse

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Retrieved on: 
Domenica, Febbraio 4, 2024
Hearing, Inflammation, Civil service commission, Knowledge, European, Cell, Haematopoiesis, COMP, IRIS, Bone marrow, Mitochondrion, Adrenal insufficiency, Regulation, Gland, ABCD1, Patient, Safety, Diagnosis, EMA, ALD, European Commission, Adrenoleukodystrophy, Seizure, B cell, PPAR, Brain, Disease, Therapy, Eurostat, Tissue, Dementia, Medicine, Committee, Hormone, DSM-IV codes, Spinal cord, Pharmaceutical industry

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Key Points: 


Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Veteran Mom Says Marine Son Was Miracle Child

Retrieved on: 
Martedì, Dicembre 5, 2023
Fisher House, Genetic testing, Meal, Travel, Adrenoleukodystrophy, Army, Hospital, Brain, ALD, Hatboro, Pennsylvania, Behavior, Transport, Diagnosis, DSM-IV codes, USS Harry S. Truman

Rockville, MD, Dec. 05, 2023 (GLOBE NEWSWIRE) -- If you talk to Army veteran Pat Sherrod-Elliott about her son, Marine Sgt.

Key Points: 
  • Rockville, MD, Dec. 05, 2023 (GLOBE NEWSWIRE) -- If you talk to Army veteran Pat Sherrod-Elliott about her son, Marine Sgt.
  • It took months at hospitals in Charleston, South Carolina and Bethesda, Maryland to get a diagnosis.
  • People born with ALD usually do not make it through childhood, which is what made Sean’s case a miracle.
  • “He loved God, loved his country, loved being a Marine, and he was a beloved father.

Poxel Provides Corporate Update and Reports Cash and Revenue for the Third Quarter and Nine Months 2023

Retrieved on: 
Mercoledì, Novembre 8, 2023
Biotechnology, Pharmaceutical, Health, Adrenoleukodystrophy, Patient, Serono, ALD, Plasma, AMN, Patent, Biomarker, Sumitomo Pharma, Diabetes, Merck, SGLT2, Jaguar 3.4 Litre, EPO, Pharmacokinetics, Male, DPP4, Disease, Prescription, Marshal of France, Pioglitazone, European Patent Office, EUR, United States patent law, Merck Serono, VLCFA, Safety, European Patent Convention, JPY, Estonia, Kidney, POC, Pharmaceutical industry, Imeglimin

Sumitomo Pharma has worked diligently to increase the production and secure inventory capacity to meet this increasing demand.

Key Points: 
  • Sumitomo Pharma has worked diligently to increase the production and secure inventory capacity to meet this increasing demand.
  • For the Sumitomo Pharma FY 2023, as a conservative assumption in line with Sumitomo Pharma’s forecast, Poxel expects to receive 8% royalties on TWYMEEG net sales.
  • On July 5th, Poxel was chosen as the winner of the 2023 edition of the I-nov contest for its program in ALD.
  • Next Financial Press Release: Fourth Quarter 2023 Cash and Revenue update, on February 15, 2024