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  2. List of drugs granted breakthrough therapy designation

    en.wikipedia.org/wiki/List_of_drugs_granted...

    CLL with 17p deletion Cabozantinib [2] Exelixis: renal cell carcinoma: Pimavanserin [2] Acadia Pharmaceuticals: Parkinson's disease-related psychosis Ibrutinib [2] Pharmacyclics: small lymphocytic lymphoma (SLL) with 17p deletion Lenvatinib [2] Eisai: renal cell carcinoma: Nivolumab [2] Bristol-Myers Squibb: Hodgkin lymphoma: Atezolizumab [2 ...

  3. List of gene therapies - Wikipedia

    en.wikipedia.org/wiki/List_of_gene_therapies

    Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11] Gendicine: treatment for head and neck squamous cell carcinoma; Idecabtagene vicleucel (Abecma): treatment for multiple myeloma [12] Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. [11] Nadofaragene firadenovec (Adstiladrin): treatment for ...

  4. Exon skipping - Wikipedia

    en.wikipedia.org/wiki/Exon_skipping

    Given these parameters, exon skipping can be used to restore an open reading frame by inducing a deletion of one or several exons within the central rod domain, and thus converting a DMD phenotype into a BMD phenotype. The genetic mutation that leads to Becker muscular dystrophy is an in-frame deletion. This means that, out of the 79 exons that ...

  5. List of genetic disorders - Wikipedia

    en.wikipedia.org/wiki/List_of_genetic_disorders

    The following is a list of genetic disorders and if known, type of mutation and for the chromosome involved. Although the parlance "disease-causing gene" is common, it is the occurrence of an abnormality in the parents that causes the impairment to develop within the child.

  6. CDKL5 deficiency disorder - Wikipedia

    en.wikipedia.org/wiki/CDKL5_deficiency_disorder

    The first medication approved to treat seizures associated with CDD is ganaxolone (Ztalmy), approved by the FDA in March 2022 [25] and by the EMA in July 2023. [26] Other efforts to develop small molecule therapeutics for CDD include phase 2 and phase 3 trials already underway or completed, and others in earlier stages of development.

  7. Antisense therapy - Wikipedia

    en.wikipedia.org/wiki/Antisense_therapy

    Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]

  8. Austin musician Casey McPherson opens Everlum Bio lab to ...

    www.aol.com/lifestyle/austin-musician-casey...

    The problem with rare diseases. A genetic mutation disease like HNRNPH2, estimated at about 150 cases globally, lacks enough patients to make finding a cure profitable for pharmaceutical companies ...

  9. Deletion (genetics) - Wikipedia

    en.wikipedia.org/wiki/Deletion_(genetics)

    The smallest single base deletion mutations occur by a single base flipping in the template DNA, followed by template DNA strand slippage, within the DNA polymerase active site. [ 3 ] [ 4 ] [ 5 ] Deletions can be caused by errors in chromosomal crossover during meiosis , which causes several serious genetic diseases .